tag:blogger.com,1999:blog-65205602651269504732024-03-14T09:20:32.509+05:30Med-ChemistMed-Chemist : "Quintessential Medicinal Chemistry"https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.comBlogger2864125tag:blogger.com,1999:blog-6520560265126950473.post-21069108635877170642024-03-14T09:20:00.001+05:302024-03-14T09:20:00.132+05:30FDA Approves Daybue (trofinetide) for the Treatment of Rett Syndrome<p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=trofinetide" target="_blank">trofinetide</a></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEi5Yd2DwN2Q7bNsno0b50AfSTjgNAyYDNDBZf-NZrmtph25DdwMJssKd7ti-ELy1TYA5cUdwUCf2A59CfHoBqjFjRbaWxPB1gHx8t2buW6djGeHbUHqXyAYsb3R3bNu8go0BOjATYphFyj0rwyDZCvqaiuICEfF02QBtaHQnAohMmzyxe-nY7gKIP7-" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="144" data-original-width="220" height="209" src="https://blogger.googleusercontent.com/img/a/AVvXsEi5Yd2DwN2Q7bNsno0b50AfSTjgNAyYDNDBZf-NZrmtph25DdwMJssKd7ti-ELy1TYA5cUdwUCf2A59CfHoBqjFjRbaWxPB1gHx8t2buW6djGeHbUHqXyAYsb3R3bNu8go0BOjATYphFyj0rwyDZCvqaiuICEfF02QBtaHQnAohMmzyxe-nY7gKIP7-" width="320" /></a></div><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced the U.S. Food and Drug Administration (FDA) approval of Daybue (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Daybue is the first and only drug approved for the treatment of Rett syndrome.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“Today marks an important milestone for the Rett community and Acadia. As the first FDA-approved drug for the treatment of Rett syndrome, Daybue now offers the potential to make meaningful differences in the lives of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by Rett syndrome,” said Steve Davis, Acadia’s Chief Executive Officer. “We are grateful to all of the Rett syndrome patients, caregivers, clinical investigators and our employees who have contributed to making today a reality and look forward to getting Daybue to patients as quickly as possible.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">“Rett syndrome is a profoundly debilitating and complex, rare, neurodevelopmental disorder that presents differently across patients and can lead to an array of unpredictable symptoms throughout the course of a patient’s life,” said Jeffrey L. Neul, M.D., Ph.D., Annette Schaffer Eskind Chair and Director, Vanderbilt Kennedy Center, Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt University Medical Center and Phase 3 LAVENDER™ study investigator. “Now, for the first time after decades of clinical research, healthcare providers finally have a treatment option to address a range of core behavioral, communication and physical symptoms for their patients living with Rett syndrome.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Rett syndrome is a complex, rare, neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene.<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2,3</span> It is characterized by a period of normal development until six to 18 months of age, followed by significant developmental regression with loss of acquired communication skills and purposeful hand use. Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities. Rett syndrome is believed to affect 6,000 to 9,000 patients in the U.S.,<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;"> </span>with a diagnosed population of approximately 4,500 U.S. patients.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The FDA approval of Daybue was supported by results from the pivotal Phase 3 LAVENDER study evaluating the efficacy and safety of trofinetide versus placebo in 187 female patients with Rett syndrome five to 20 years of age. In the study, treatment with Daybue demonstrated statistically significant improvement compared to placebo on both co-primary efficacy endpoints, as measured by the change from baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) total score (p=0.018) and the Clinical Global Impression-Improvement (CGI-I) scale score (p=0.003) at week 12. The RSBQ is a caregiver assessment that evaluates a range of symptoms of Rett syndrome including vocalizations, facial expressions, eye gaze, hand movements (or stereotypies), repetitive behaviors, breathing, night-time behaviors and mood. The CGI-I is a global physician assessment of whether a patient has improved or worsened. In the study, the most common side effects were diarrhea (82%) and vomiting (29%).</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“This is a historic day for the Rett syndrome community and a meaningful moment for the patients and caregivers who have eagerly awaited the arrival of an approved treatment for this condition,” said Melissa Kennedy, Chief Executive Officer of the International Rett Syndrome Foundation. “Rett syndrome is a complicated, devastating disease that affects not only the individual patient, but whole families. With today’s FDA decision, those impacted by Rett have a promising new treatment option that has demonstrated benefit across a variety of Rett symptoms, including those that impact the daily lives of those living with Rett and their loved ones.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-daybue-trofinetide-rett-syndrome-5983.html" style="background-color: transparent;">FDA Approves Daybue (trofinetide) for the Treatment of Rett Syndrome<br /></a></p><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-31891650692760601862024-03-11T09:26:00.002+05:302024-03-11T09:26:00.125+05:30FDA Approves Zavzpret (zavegepant) Nasal Spray for the Acute Treatment of Migraine<div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Pfizer Inc. (NYSE: PFE) announced the U.S. Food and Drug Administration (FDA) approval of Zavzpret (zavegepant), the first and only calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray for the acute treatment of migraine with or without aura in adults. In its pivotal Phase 3 study, Zavzpret was statistically superior to placebo on the co-primary endpoints of pain freedom and freedom from most bothersome symptom at two hours post-dose. The pivotal study also demonstrated pain relief as early as 15 minutes in a prespecified secondary endpoint versus placebo.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEjjZhYy28fB1g5Qsw1ZrGlj2F0ZQwtjtgP1yKn9wk10mVMSPkVTYwX0IoSlO-xTYoInTEp-8vADu1bufZ43yLp6SWfklrW8VeBzghtu5lbJAtVePe4WVmPUqIyIHS94Gwpwvqe72jKCHr0Vrd7bbYNf4WvDcMIqGL1beFEHGjEKy29OQeLdKV-bO-0T" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="752" data-original-width="800" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEjjZhYy28fB1g5Qsw1ZrGlj2F0ZQwtjtgP1yKn9wk10mVMSPkVTYwX0IoSlO-xTYoInTEp-8vADu1bufZ43yLp6SWfklrW8VeBzghtu5lbJAtVePe4WVmPUqIyIHS94Gwpwvqe72jKCHr0Vrd7bbYNf4WvDcMIqGL1beFEHGjEKy29OQeLdKV-bO-0T" width="255" /></a></div><br /><br /><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“The FDA approval of Zavzpret marks a significant breakthrough for people with migraine who need freedom from pain and prefer alternative options to oral medications,” said Angela Hwang, Chief Commercial Officer, President, Global Biopharmaceuticals Business, Pfizer. “Zavzpret underscores Pfizer’s commitment to delivering an additional treatment option to help people with migraine gain relief and get back to their daily lives. Pfizer will continue to build its migraine franchise to further support the billions of people worldwide impacted by this debilitating disease.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The FDA approval is based on two pivotal randomized, double-blind, placebo-controlled studies that established the efficacy, tolerability and safety profiles of Zavzpret for the acute treatment of migraine. In these studies, Zavzpret was statistically superior to placebo on the co-primary endpoints of pain freedom (defined as a reduction of moderate or severe headache pain to no headache pain) and freedom from most bothersome symptom at two hours post-dose (defined as the absence of the self-identified most bothersome symptom). The pivotal Phase 3 study published in <em style="box-sizing: inherit;">The Lancet Neurology</em> found Zavzpret showed broad efficacy by also demonstrating statistically significant superiority to placebo across 13 of 17 prespecified secondary outcome measures, including early time point endpoints (e.g., 15 and 30-minute pain relief and return to normal function at 30 minutes), return to normal function at 2 hours, and durable efficacy endpoints (e.g., 2-24 and 2-48 hour sustained pain freedom and sustained pain relief). On the 14th endpoint, return to normal function at 15 minutes post-dose, the difference between Zavzpret and placebo was not significant. Consequently, in keeping with the trial’s statistical analysis plan, the remaining secondary endpoints were not formally tested.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">“When a migraine hits, it has a significant negative impact on a person’s daily life,” said Kathleen Mullin, M.D., Associate Medical Director at New England Institute for Neurology & Headache. “Among my migraine patients, one of the most important attributes of an acute treatment option is how quickly it works. As a nasal spray with rapid drug absorption, Zavzpret offers an alternative treatment option for people who need pain relief or cannot take oral medications due to nausea or vomiting, so they can get back to normal function quickly.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><p><span style="text-align: left;">Zavzpret was well tolerated in clinical trials. The most common adverse reactions reported in at least 2% of patients treated with Zavzpret and at a frequency greater than placebo were taste disorders (includes dysgeusia and ageusia), nausea, nasal discomfort and vomiting. Zavzpret is contraindicated in patients with a history of hypersensitivity to zavegepant or to any of its components. Hypersensitivity reactions, including facial swelling and urticaria, have occurred with Zavzpret in clinical studies.</span> </p><p> https://en.wikipedia.org/wiki/Zavegepant#/media/File:Zavegepant.svg</p><p></p></div><div style="text-align: justify;">https://en.wikipedia.org/wiki/Zavegepant</div><div><br /></div><div><br /></div><a href="https://www.drugs.com/newdrugs/fda-approves-zavzpret-zavegepant-nasal-acute-migraine-5982.html">FDA Approves Zavzpret (zavegepant) Nasal Spray for the Acute Treatment of Migraine</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-2382273296867454272024-03-08T09:30:00.001+05:302024-03-08T09:30:00.126+05:30FDA Approves Skyclarys (omaveloxolone) for the Treatment of Friedreich’s Ataxia<div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=omaveloxolone" target="_blank">omaveloxolone</a></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEgoRhfYyfng59KsSo6xZ_PuiSZONA_0L2nNT5bVlCxiSmrxyhVqNHcsF_avHgk4crdfgtHNsUhMUPV0Xm1QLi0xl4xTtAeCib2J3WkFA7Agy3In7DRzGVO2CWqzVMH8JlT2z-21mwN_kd5u5Vo1MBmUgRgp1_Hm2MgZce7zZpPUIvX_1runIi1d6DfC" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="156" data-original-width="250" height="200" src="https://blogger.googleusercontent.com/img/a/AVvXsEgoRhfYyfng59KsSo6xZ_PuiSZONA_0L2nNT5bVlCxiSmrxyhVqNHcsF_avHgk4crdfgtHNsUhMUPV0Xm1QLi0xl4xTtAeCib2J3WkFA7Agy3In7DRzGVO2CWqzVMH8JlT2z-21mwN_kd5u5Vo1MBmUgRgp1_Hm2MgZce7zZpPUIvX_1runIi1d6DfC" width="320" /></a></div><br /><br /><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Reata Pharmaceuticals, Inc. (Nasdaq: RETA) announced the U.S. Food and Drug Administration (“FDA”) approval of Skyclarys™ (omaveloxolone) for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. With this approval, the FDA granted a rare pediatric disease priority review voucher.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">"The approval of Skyclarys, the first therapy specifically indicated for the treatment of Friedreich’s ataxia, is an important milestone for patients affected by this disease as well as their families and caregivers," said Warren Huff, Reata's Chief Executive Officer. "We are grateful to Friedreich’s ataxia patients, investigators, U.S. regulators, and our scientists and employees who made this approval possible. As a company, this is a transformative milestone that highlights our commitment to developing and commercializing novel therapies for patients with severe diseases with few or no approved therapies. We look forward to delivering Skyclarys to eligible patients as quickly as possible."</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p></blockquote><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Friedreich’s ataxia is an ultra-rare, inherited neurodegenerative disorder that is typically diagnosed during adolescence. Patients with Friedreich’s ataxia experience progressive loss of coordination, muscle weakness, and fatigue, which commonly progresses to motor incapacitation and wheelchair reliance by their teens or early twenties, and eventually death. Friedreich’s ataxia affects approximately 5,000 diagnosed patients in the U.S.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">“Friedreich's ataxia is a debilitating neuromuscular disease that progressively robs patients of their mobility and independence,” said Susan Perlman, MD, Clinical Professor, Department of Neurology, David Geffen School of Medicine, UCLA. “The approval of Skyclarys represents an important step forward in the treatment of Friedreich's ataxia, providing physicians with the first disease-specific treatment option approved for patients living with this ultra-rare and progressive disease.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">"Today’s approval of Skyclarys represents a significant milestone in our effort to advance research and achieve treatments for Friedreich’s ataxia," said Jen Farmer, Chief Executive Officer at Friedreich's Ataxia Research Alliance. "The entire Friedreich's ataxia community including patients, clinicians, scientists, pharmaceutical companies, government agencies, and others have worked collaboratively for decades to enable therapeutic development for this debilitating disease. Today, we celebrate the impact of an engaged patient community, and we are grateful to the FDA and Reata for working together on the approval of Skyclarys, the first therapy approved in the United States for adult and adolescent patients aged 16 years and older with Friedreich's ataxia.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The approval of Skyclarys is supported by the efficacy and safety data from the MOXIe Part 2 trial and a post hoc Propensity-Matched Analysis of the open-label MOXIe Extension trial.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">MOXIe Part 2 was a randomized, double-blind, placebo-controlled study. Patients with genetically confirmed Friedreich’s ataxia and baseline modified Friedreich’s Ataxia Rating Scale (“mFARS”) scores between 20 and 80 were randomized 1:1 to receive placebo or 150 mg of Skyclarys daily. The primary endpoint was change from baseline in mFARS score compared to placebo at Week 48 in the Full Analysis Population of patients without severe pes cavus (n=82). The mFARS is a clinical assessment tool to assess patient function and is used in clinical trials to assess the efficacy of investigational products for use in Friedreich’s ataxia. Treatment with Skyclarys resulted in statistically significant lower mFARS scores (less impairment) relative to placebo at Week 48. The placebo-corrected difference between the two groups was -2.41 points with a p-value of 0.0138. The most common adverse reactions in MOXIe Part 2 (≥20% and greater than placebo) were elevated liver enzymes (AST/ALT), headache, nausea, abdominal pain, fatigue, diarrhea, and musculoskeletal pain.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Further, in a post hoc Propensity-Matched Analysis, mFARS progression of patients treated with 150 mg of Skyclarys daily in the open-label MOXIe Extension trial was compared to the progression of propensity score-matched untreated patients in the largest natural history study of Friedreich’s ataxia, Clinical Outcome Measures in Friedreich’s ataxia (“FA-COMS”). All patients enrolled in the MOXIe Extension study with at least one post-baseline assessment (n=136) were matched one to one with patients from the FA-COMS study (n=136). Lower (improved) mFARS scores were observed in patients treated with Skyclarys after 3 years relative to the matched set of untreated patients from the FA-COMS natural history study. These exploratory analyses should be interpreted cautiously given the limitations of data collected outside of a controlled study, which may be subject to confounding.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><br /></p></div><a href="https://www.drugs.com/newdrugs/fda-approves-skyclarys-omaveloxolone-friedreich-s-ataxia-5979.html">FDA Approves Skyclarys (omaveloxolone) for the Treatment of Friedreich’s Ataxia</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-44083538183630607212024-03-06T09:12:00.001+05:302024-03-06T09:12:00.132+05:30FDA Approves Rezzayo (rezafungin for injection) for the Treatment of Candidemia and Invasive Candidiasis<div style="text-align: justify;"><br /></div><div style="text-align: justify;"><span style="background-color: white; color: #202227;"><span style="font-family: Playfair Display;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=rezafungin" target="_blank">rezafungin</a></span></span></div><div style="text-align: justify;"><span style="background-color: white; color: #202227;"><br /></span></div><div style="text-align: justify;"><span style="background-color: white; color: #202227;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEhIxNQly2uLssKVssUByopTi18r0zUOFLpaZ4ZiZRCz4-iPC2soHqJ5B_MQlTT6YdFNANd3SWYW4tx2BatVpApxHCgYi9szI7fF_iam-vdcAzj2gAm3IoFEVh3LVIHDDsMfDTD3kSFLGnfAH2Jc6BTXN2TgU78fqcxP-WnJ4a8ziTeoT4pJtbdpv18Q" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="387" data-original-width="497" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEhIxNQly2uLssKVssUByopTi18r0zUOFLpaZ4ZiZRCz4-iPC2soHqJ5B_MQlTT6YdFNANd3SWYW4tx2BatVpApxHCgYi9szI7fF_iam-vdcAzj2gAm3IoFEVh3LVIHDDsMfDTD3kSFLGnfAH2Jc6BTXN2TgU78fqcxP-WnJ4a8ziTeoT4pJtbdpv18Q" width="308" /></a></div><br /><br /></span></div><h2 class="ddc-media-title" style="-webkit-font-smoothing: antialiased; background-color: white; box-sizing: inherit; color: #202227; letter-spacing: -0.01em; line-height: 1.3; margin: 0px 0px 8px;"><span style="text-align: justify;"><span style="font-family: Playfair Display; font-size: small;"><br /></span></span></h2><h2 class="ddc-media-title" style="-webkit-font-smoothing: antialiased; background-color: white; box-sizing: inherit; color: #202227; letter-spacing: -0.01em; line-height: 1.3; margin: 0px 0px 8px;"><span style="font-family: Playfair Display; font-size: small; font-weight: normal;"><span style="text-align: justify;">Cidara Therapeutics, Inc. and Melinta Therapeutics, LLC announced the U.S. Food and Drug Administration (FDA) approval of </span><span style="text-align: justify;">Rezzayo</span><span style="text-align: justify;"> </span><span style="text-align: justify;">(rezafungin for injection) for the treatment of candidemia and invasive candidiasis in adults with limited or no alternative treatment options. Rezzayo is the first new treatment option approved for patients with candidemia and invasive candidiasis in over a decade.</span></span></h2><div><blockquote style="background-color: white; border-left: 6px solid var(--color-border-subtle); box-sizing: inherit; color: #202227; font-style: italic; margin: 0px 0px 24px; padding-left: 20px;"><p id="pull-quote" style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px;"></p></blockquote><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p></div><blockquote><div><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“The FDA approval of Rezzayo represents a significant milestone for Cidara, and for patients confronted with difficult-to-treat and often deadly candidemia and invasive candidiasis,” said Jeffrey Stein, Ph.D., president and chief executive officer of Cidara. “I am extremely proud of all of the Cidara employees who collectively advanced Rezzayo from preclinical development to NDA approval and am grateful to the many patients and healthcare teams who have participated in the clinical studies.”</span></p></div><div></div></blockquote><div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">George Thompson, M.D., principal investigator in the ReSTORE trial and professor of clinical medicine at the University of California, Davis, School of Medicine, added, “The FDA approval of Rezzayo is tremendous news for those of us who have been hoping for a new option to treat our patients with these deadly fungal infections. Based on the totality of clinical data generated, Rezzayo has the potential to simplify the management of invasive candidiasis and enhance the continuity of echinocandin care.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The FDA approval of once-weekly Rezzayo was based on clinical data from Cidara’s global ReSTORE Phase 3 trial and supported by the STRIVE Phase 2 clinical trial and extensive non-clinical development program. In clinical studies, Rezzayo, dosed once-weekly, met the FDA and EMA primary endpoints, demonstrating statistical non-inferiority versus caspofungin, a current once-daily standard of care. In addition, overall rates of adverse events and serious adverse events were comparable in patients receiving Rezzayo and caspofungin, while rates of adverse events leading to study drug discontinuation were also similar for Rezzayo and caspofungin. Based on Qualified Infectious Disease Product (QIDP) designation, Rezzayo was approved under Priority Review.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Christine Ann Miller, president and chief executive officer of Melinta Therapeutics, added, “We are thrilled that the FDA has approved Rezzayo, and are firmly committed to offering this innovative therapy to address unmet medical needs and simplify the treatment for patients suffering from invasive <em style="box-sizing: inherit;">Candida</em> infections. We intend to leverage our expansive commercial infrastructure and experience launching anti-infective drugs into acute care settings. We are working closely with Cidara and anticipate bringing Rezzayo, a differentiated once-weekly treatment to patients, this summer.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Last year, Melinta announced that it had acquired the exclusive rights to commercialize Rezzayo in the U.S. from Cidara. Cidara retains the rights to rezafungin in Japan and has licensed the commercial rights to Melinta Therapeutics in the U.S. and Mundipharma in all other geographies. The European Medicines Agency (EMA) accepted the marketing authorization application (MAA) for rezafungin in August 2022 and it is currently under review.</p></div><div><span style="font-family: "Playfair Display";">https://en.wikipedia.org/wiki/Rezafungin</span></div><div><span style="font-family: Playfair Display;"><br /></span></div><div><br /></div><a href="https://www.drugs.com/newdrugs/fda-approves-rezzayo-rezafungin-candidemia-invasive-candidiasis-5989.html"><span style="font-family: Playfair Display;">FDA Approves Rezzayo (rezafungin for injection) for the Treatment of Candidemia and Invasive Candidiasis</span></a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-1670131120170174462024-03-04T10:46:00.001+05:302024-03-04T10:46:00.130+05:30FDA Approves Brenzavvy (bexagliflozin) for the Treatment of Adults with Type 2 Diabetes<div><div class="bw-release-timestamp" style="background-color: white; box-sizing: inherit; color: #202227; margin: 0px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">TheracosBio announced the U.S. Food and Drug Administration (FDA) approval of Brenzavvy (bexagliflozin), an oral sodium-glucose cotransporter 2 (SGLT2) inhibitor. Brenzavvy is indicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. Brenzavvy is not recommended for patients with type 1 diabetes mellitus or for the treatment of diabetic ketoacidosis. Brenzavvy is contraindicated in patients who are hypersensitive to bexagliflozin or any tablet ingredient and is not indicated for the treatment of type 2 diabetes in patients with end stage renal disease or who are receiving dialysis.</span></div><div class="bw-release-story" style="background-color: white; box-sizing: inherit; color: #202227; margin: 0px; padding: 0px;"><blockquote style="border-left: 6px solid var(--color-border-subtle); box-sizing: inherit; font-style: italic; margin: 0px 0px 24px; padding-left: 20px;"><p id="pull-quote" style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px;"></p></blockquote><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">The FDA approval is based on results from a clinical program that evaluated the safety and efficacy of Brenzavvy in 23 clinical trials enrolling more than 5,000 adults with type 2 diabetes mellitus. Phase 3 studies showed Brenzavvy significantly reduced hemoglobin A1c and fasting blood sugar after 24 weeks, either as a monotherapy, in combination with metformin, or as an add-on to standard-of-care treatment consisting of a variety of regimens, including metformin, sulfonylureas, insulin, DPP4 inhibitors, or combinations of these agents. Although Brenzavvy is not approved for weight or blood pressure reduction, modest decreases in both weight and systolic blood pressure have been observed in the clinical program.</span></p></div></div><div style="text-align: justify;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEgu0LSaVFG3BFaHDaLTcUK6mJdT-GVpwGS5LFApKg4EhwZxrXv0EQCjTa8NRcv0qZEZ3t09X6fZUShLtYCa0cIeu4o3TYr32thDpRO_SlVqc77PExcSC12eOjVH9DIAFdaHk_cQ0M0RmhY2R3cHN2Uq1bF1EgqVddUvFxxKrmCxFMKLpPzguwwBC0Rp" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="444" data-original-width="1024" height="139" src="https://blogger.googleusercontent.com/img/a/AVvXsEgu0LSaVFG3BFaHDaLTcUK6mJdT-GVpwGS5LFApKg4EhwZxrXv0EQCjTa8NRcv0qZEZ3t09X6fZUShLtYCa0cIeu4o3TYr32thDpRO_SlVqc77PExcSC12eOjVH9DIAFdaHk_cQ0M0RmhY2R3cHN2Uq1bF1EgqVddUvFxxKrmCxFMKLpPzguwwBC0Rp" width="320" /></a></div><br /><br /></div><div style="text-align: justify;"><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">“As a class of drugs, SGLT2 inhibitors have shown tremendous benefit in treating adults with type 2 diabetes,” said Dr. Mason Freeman, M.D., Director of the Translational Research Center at Massachusetts General Hospital. “Being involved in all of the clinical trials for Brenzavvy, I am greatly impressed with the efficacy of the drug in reducing blood glucose levels and I believe it is an important addition to the SGLT2 inhibitor class of drugs.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Brenzavvy treatment can be initiated in adults with type 2 diabetes with an estimated glomerular filtration rate (eGFR) greater than 30 mL/min/1.73 m<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2</span>. Patients with eGFR less than 60 and greater than 30 mL/min/1.73 m<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2</span> are said to be in stage 3 chronic kidney disease, and for these patients metformin is often avoided due to the risk of lactic acidosis.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“Today's FDA approval represents a significant milestone for TheracosBio and provides an important treatment option to patients who suffer from type 2 diabetes. We look forward to bringing Brenzavvy to market,” said Albert R. Collinson, Ph.D., President and CEO of TheracosBio. “The approval of the Brenzavvy NDA is a result of the tireless work of the TheracosBio team and investigators. I want to thank all of the patients who took part in our clinical trials.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">According to the U.S. Centers for Disease Control and Prevention, more than 33 million Americans have type 2 diabetes, which means their bodies don’t use insulin correctly and as a result their blood sugar levels are too high. While some people can control their blood sugar levels with exercise and a healthy diet, others may need additional help to achieve good blood sugar (glycemic) control.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">SGLT2 inhibitors are a class of prescription medicines that lower blood sugar by causing the kidneys to remove sugar from the body through urine.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Brenzavvy is available as 20 mg oral tablets recommended to be taken once daily, in the morning with or without food.</p></div><div><br /></div><div>https://en.wikipedia.org/wiki/Bexagliflozin</div><div><br /></div><a href="https://www.drugs.com/newdrugs/fda-approves-brenzavvy-bexagliflozin-adults-type-2-diabetes-5960.html">FDA Approves Brenzavvy (bexagliflozin) for the Treatment of Adults with Type 2 Diabetes</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-80497073098431016392024-03-01T11:03:00.001+05:302024-03-01T11:03:00.133+05:30FDA Approves Jaypirca (pirtobrutinib) for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma<div style="text-align: justify;"><br /></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;">Loxo@Lilly, the oncology unit of Eli Lilly and Company (NYSE: LLY), announced the U.S. Food and Drug Administration (FDA) approved </span>Jaypirca<span style="background-color: white; color: #202227;">™ (pirtobrutinib, 100 mg & 50 mg tablets) for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a Bruton's tyrosine kinase (BTK) inhibitor. Jaypirca was approved under the FDA's Accelerated Approval pathway based on response rate from the open-label, single-arm, international, Phase 1/2 study, called the BRUIN trial.</span><span style="background-color: white; box-sizing: inherit; color: #202227; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1</span><span style="background-color: white; color: #202227;"> Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.</span></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;"><br /></span></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEi6Ek2BX1qv5kOwgmbq8lGHMLIXUXQPO4nuNC4b_Ouw55sk8mPZ0UTSPkjOBKe_DIMku_9hWAxi0RlclSS7x_fV2kYVtIvQmDigaORitedgRl-X3O6lIeaUMKbslM-vsBqVp8mdxjfHUbysoP-wVohkcjTE9tDny-8ZTG9nCwQVZNPSwtM6TDORwz6K" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="506" data-original-width="1024" height="158" src="https://blogger.googleusercontent.com/img/a/AVvXsEi6Ek2BX1qv5kOwgmbq8lGHMLIXUXQPO4nuNC4b_Ouw55sk8mPZ0UTSPkjOBKe_DIMku_9hWAxi0RlclSS7x_fV2kYVtIvQmDigaORitedgRl-X3O6lIeaUMKbslM-vsBqVp8mdxjfHUbysoP-wVohkcjTE9tDny-8ZTG9nCwQVZNPSwtM6TDORwz6K" width="320" /></a></div><br /><br /></span></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></div><div><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Jaypirca, a highly selective kinase inhibitor, utilizes a novel binding mechanism and is the first and only FDA approved non-covalent (reversible) BTK inhibitor. Jaypirca can reestablish BTK inhibition in MCL patients previously treated with a covalent BTK inhibitor (ibrutinib, acalabrutinib, or zanubrutinib) and extend the benefit of targeting the BTK pathway.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">"The approval of Jaypirca represents an important advance for patients with relapsed or refractory MCL, who currently have limited options and historically have had a poor prognosis following discontinuation of treatment with a covalent BTK inhibitor," said <span class="xn-person" style="box-sizing: inherit;">Michael Wang</span>, M.D., Puddin Clarke Endowed Professor of Lymphoma and Myeloma at The <span class="xn-org" style="box-sizing: inherit;">University of Texas</span> MD Anderson Cancer Center. "These data indicate that Jaypirca can provide efficacy in patients previously treated with a covalent BTK inhibitor, potentially extending the time patients may benefit from BTK inhibition therapy. Jaypirca offers a new approach to targeting the BTK pathway following treatment with a covalent BTK inhibitor and has the potential to meaningfully impact the treatment paradigm for relapsed and refractory MCL patients."</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">The labeling for Jaypirca contains warnings and precautions for infections, hemorrhage, cytopenias, atrial fibrillation and flutter, second primary malignancies, and embryo-fetal toxicity. See Important Safety Information below and full Prescribing Information for additional information, including dosing modifications.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">"We are pleased to bring a meaningful new therapeutic option to patients with MCL that can reestablish the benefit of targeting the BTK pathway after receiving multiple prior therapies, including a covalent BTK inhibitor," said <span class="xn-person" style="box-sizing: inherit;">Jacob Van Naarden</span>, chief executive officer, Loxo@Lilly. "We are grateful to the patients, investigators, and other members of the clinical care teams for their contributions. Our team has been committed to rapidly advancing the development of Jaypirca for patients with MCL, and we look forward to building on this milestone by continuing to bring forward important new treatments for people with hematologic malignancies."</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">The FDA approval is based on data from a subset of patients in the BRUIN Phase 1/2 trial. The assessment of efficacy was based on 120 patients with MCL treated with Jaypirca 200 mg once daily until disease progression or unacceptable toxicity. Patients with active central nervous system lymphoma or allogeneic hematopoietic stem cell transplantation or CAR T-cell therapy within 60 days were excluded. Patients had received a median of three prior lines of therapy (range: 1 to 9), with 93% having two or more prior lines; all patients received one or more prior lines of therapy containing a covalent BTK inhibitor. Eighty-three percent (83%) of patients discontinued their last BTK inhibitor due to refractory or progressive disease. Efficacy was based on overall response rate (ORR) and duration of response (DOR) as assessed by an independent review committee (IRC) using 2014 Lugano criteria.</span></p></div><div><a href="https://www.drugs.com/newdrugs/fda-approves-jaypirca-pirtobrutinib-adult-patients-relapsed-refractory-mantle-cell-lymphoma-5963.html" target="_blank">Read More </a></div><div><br /></div><div>https://en.wikipedia.org/wiki/Pirtobrutinib#/media/File:Pirtobrutinib.svg</div><div><br /></div><a href="https://www.drugs.com/newdrugs/fda-approves-jaypirca-pirtobrutinib-adult-patients-relapsed-refractory-mantle-cell-lymphoma-5963.html">FDA Approves Jaypirca (pirtobrutinib) for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-8857872033663074622024-02-29T12:54:00.001+05:302024-02-29T12:54:00.127+05:30FDA Approves Orserdu (elacestrant) for Patients with ESR1 Mutations in ER+, HER2- Advanced or Metastatic Breast Cancer<div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;">The Menarini Group (“Menarini”), a leading Italian pharmaceutical and diagnostics company, announced the U.S. Food and Drug Administration (FDA) approval of </span>Orserdu<span style="background-color: white; color: #202227;"> for the treatment of postmenopausal women or adult men, with ER+, HER2-, </span><em style="background-color: white; box-sizing: inherit; color: #202227;">ESR1</em><span style="background-color: white; color: #202227;">-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. Stemline Therapeutics (“Stemline”), a wholly-owned subsidiary of the Menarini Group, headquartered in New York and focused on bringing transformational oncology treatments for cancer patients, will commercialize Orserdu in the U.S.</span></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;"><br /></span></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEgClZPN0krEqADC_n1cQd9AIH_lKOqSLlarN4xOSpFh_8AbK5L5AqP_uaLnoLF0GErbs0dmS0eOmPSzBB5Apz9aUTGR17qTtjxCxk6bPUBPpf7gBN72T5E76IW2xMcubXQpedoHxn1q0_XEpPOBaHjnQBrCe3DC3enJoSrMNMZa6CDwajF_7ymkeQws" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="917" data-original-width="800" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEgClZPN0krEqADC_n1cQd9AIH_lKOqSLlarN4xOSpFh_8AbK5L5AqP_uaLnoLF0GErbs0dmS0eOmPSzBB5Apz9aUTGR17qTtjxCxk6bPUBPpf7gBN72T5E76IW2xMcubXQpedoHxn1q0_XEpPOBaHjnQBrCe3DC3enJoSrMNMZa6CDwajF_7ymkeQws" width="209" /></a></div><br /><br /></span></span></div><div style="text-align: justify;"><span style="background-color: white; color: #202227;"><span style="font-family: Playfair Display;"><br /></span></span></div><div style="text-align: justify;"><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“The FDA approval of Orserdu marks the first ever therapy for ER+, HER2- advanced or metastatic breast cancer patients with <em style="box-sizing: inherit;">ESR1</em> mutations and we are very proud to offer a targeted therapy addressing this huge unmet need,” commented Elcin Barker Ergun, Chief Executive Officer of the Menarini Group. “We are grateful to the patients, investigators and administrators who participated in the clinical trials that led to this remarkable innovation.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Orserdu is approved under the FDA’s Priority Review and Fast Track designation based on the results of the registrational Phase III trial EMERALD, that demonstrated statistically significant progression-free survival (PFS) with elacestrant vs SOC endocrine monotherapy (fulvestrant, letrozole, anastrozole, exemestane), meeting both primary endpoints in all patients and in those patients whose tumors harbor <em style="box-sizing: inherit;">ESR1</em> mutations.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">In the group of patients whose tumors had <em style="box-sizing: inherit;">ESR1</em> mutations, elacestrant reduced the risk of progression or death by 45% (PFS HR=0.55, 95% CI: 0.39, 0.77) vs SOC. A post-hoc analysis of the PFS results based on the duration of prior CDK4/6i inhibitors (CDK4/6i) usage was presented at San Antonio Breast Cancer Symposium (SABCS) in December 2022. The median PFS was 8.6 months on elacestrant vs 1.9 months for SOC, in those patients whose tumors harbored <em style="box-sizing: inherit;">ESR1</em> mutations and had been treated with a CDK4/6i for at least 12 months.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Safety data is consistent with the other endocrine therapies. Most of the adverse events (AEs), including nausea and musculoskeletal pain were grade 1 and 2. No hematological safety signal was observed and none of the patients in either of the two treatment arms had sinus bradycardia.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“Advanced or metastatic <span lang="EN-US" style="box-sizing: inherit;">E</span>R+, HER2- breast cancer pre-treated with endocrine-based therapy remains an area of unmet medical need. The last endocrine therapy approved was about 20 years ago, and effective endocrine options for this patient population are needed<span lang="EN-US" style="box-sizing: inherit;">,</span>” said Dr. Aditya Bardia, MD, MPH, Director of Breast Cancer Research at Mass General Cancer Center, Associate Professor at the Medicine Department at Harvard Medical School, and Principal Investigator for the EMERALD trial. “<em style="box-sizing: inherit;">ESR1</em><em style="box-sizing: inherit;"> </em><span lang="EN-US" style="box-sizing: inherit;">mutations are</span> a known driver of resistance to standard endocrine therapy, and so far, ha<span lang="EN-US" style="box-sizing: inherit;">ve</span> been difficult to treat. <span lang="EN-US" style="box-sizing: inherit;">The </span>approval <span lang="EN-US" style="box-sizing: inherit;">of elacestrant </span>is welcome<span lang="EN-US" style="box-sizing: inherit;">d</span> as it offers a novel option for patients with <span lang="EN-US" style="box-sizing: inherit;">E</span>R+, HER2- metastatic breast cancer. This therapy target<span lang="EN-US" style="box-sizing: inherit;">s</span> <span lang="EN-US" style="box-sizing: inherit;">the </span><em style="box-sizing: inherit;">ESR1</em> <span lang="EN-US" style="box-sizing: inherit;">mutations in </span>metastatic breast cancer<span lang="EN-US" style="box-sizing: inherit;"> and provides patients</span> with a convenient oral once-daily dose.<span lang="EN-US" style="box-sizing: inherit;">”</span></span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“Each year 300,000 Americans are diagnosed with breast cancer and metastatic breast cancer causes the vast majority of deaths from the disease: more than 43,000 annually. We urgently need new and better treatment options to extend and improve the lives of people with metastatic breast cancer,” said Sonya Negley, Executive Director, Metavivor. “We are thrilled to see the approval of Orserdu, a new oral endocrine therapy, for patients who have tumors that harbor <em style="box-sizing: inherit;">ESR1</em> mutations, which are present in up to 40% of ER+, HER2- advanced or metastatic breast cancer. We advise patients to get tested for <em style="box-sizing: inherit;">ESR1</em> mutations at progression in their metastatic treatment, so that their healthcare team can identify the right treatment options for their disease.“</span></p><div><a href="https://www.drugs.com/newdrugs/fda-approves-orserdu-elacestrant-patients-esr1-mutations-er-her2-advanced-metastatic-breast-cancer-5964.html" target="_blank">Read More ...</a></div><div><br /></div></div><div style="text-align: justify;"><span style="background-color: white; font-size: 17px;"><span style="color: #202227; font-family: -apple-system, BlinkMacSystemFont, Segoe UI, Segoe UI Symbol, Helvetica Neue, Roboto, Arial, sans-serif;">https://en.wikipedia.org/wiki/Elacestrant</span></span></div><div style="text-align: justify;"><span style="background-color: white; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;"><br /></span></div><div><a href="https://www.drugs.com/newdrugs/fda-approves-orserdu-elacestrant-patients-esr1-mutations-er-her2-advanced-metastatic-breast-cancer-5964.html">FDA Approves Orserdu (elacestrant) for Patients with ESR1 Mutations in ER+, HER2- Advanced or Metastatic Breast Cancer</a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-1953589119568614852024-02-27T13:07:00.001+05:302024-02-27T13:07:00.127+05:30FDA Approves Jesduvroq (daprodustat) for Anemia Caused by Chronic Kidney Disease for Adults on Dialysis<div><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">GSK plc (LSE/NYSE: GSK) announced the US Food and Drug Administration (FDA) approval of Jesduvroq (daprodustat), an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), for the once-a-day treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least four months. <em style="box-sizing: inherit;">Jesduvroq</em> is the first innovative medicine for anemia treatment in over 30 years and the only HIF-PHI approved in the US, providing a new oral, convenient option for patients in the US with anemia of CKD on dialysis.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEjoXPGqKDMr4xcmW-AkdmRNhHKhp_ZxdwJnCXF9EVAjLIly_ipRIGOLl7Tizsha5NSr7LENMQjWrTacSGs3FvvuMZPYhQ4Su29M-0W2Nw1RaMfZEY7sFHNvLVLzKieAkdfVauN5iBipDsW5Nmf9j7rlV6Nyt6CXfpCFkPt2SjCaKJg4El-Djvt6onMR" style="margin-left: 1em; margin-right: 1em;"><span style="font-family: inherit;"><img alt="" data-original-height="570" data-original-width="800" height="228" src="https://blogger.googleusercontent.com/img/a/AVvXsEjoXPGqKDMr4xcmW-AkdmRNhHKhp_ZxdwJnCXF9EVAjLIly_ipRIGOLl7Tizsha5NSr7LENMQjWrTacSGs3FvvuMZPYhQ4Su29M-0W2Nw1RaMfZEY7sFHNvLVLzKieAkdfVauN5iBipDsW5Nmf9j7rlV6Nyt6CXfpCFkPt2SjCaKJg4El-Djvt6onMR" width="320" /></span></a></div><span style="font-family: inherit;"><br /><br /></span><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;"><br /></span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">The FDA approval is based on results from the ASCEND-D trial, assessing the efficacy and safety of <em style="box-sizing: inherit;">Jesduvroq</em> for the treatment of anemia of CKD in patients on dialysis. Results were published in the <em style="box-sizing: inherit;">New England Journal of Medicine</em> with additional results published in the <em style="box-sizing: inherit;">New England Journal of Medicine</em> supplementary appendix.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">The Safety Information for <em style="box-sizing: inherit;">Jesduvroq </em>includes a boxed warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, and thrombosis of vascular access. <em style="box-sizing: inherit;">Jesduvroq</em> increases the risk of thrombotic vascular events, including major adverse cardiovascular events (MACE). Targeting a hemoglobin level greater than 11 g/dL is expected to further increase the risk of death and arterial venous thrombotic events, as occurs with erythropoietin stimulating agents (ESAs), which also increase erythropoietin levels. No trial has identified a hemoglobin target level, dose of <em style="box-sizing: inherit;">Jesduvroq</em>, or dosing strategy that does not increase these risks. Use the lowest dose of <em style="box-sizing: inherit;">Jesduvroq</em> sufficient to reduce the need for red blood cell transfusions. <em style="box-sizing: inherit;">Jesduvroq</em> has not been shown to improve quality of life, fatigue, or patient well-being. <em style="box-sizing: inherit;">Jesduvroq</em> is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia or for treatment of anemia of chronic kidney disease in patients who are not on dialysis.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">CKD is an increasing global health burden affecting 700 million patients worldwide, with an estimated one in seven patients also developing anemia.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1,</span><span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2</span> When left untreated or undertreated, anemia of CKD is associated with poor clinical outcomes and leads to a substantial burden on patients and healthcare systems.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">3</span> There is an unmet need for oral treatment options with efficacy and safety comparable to current treatments.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;"><span style="box-sizing: inherit; font-weight: 600;">LaVarne Burton, President and Chief Executive Officer, American Kidney Fund, said: </span><em style="box-sizing: inherit;">“</em>Anemia of CKD can be a debilitating condition that is challenging to manage. This news means that patients on dialysis who are living with anemia of CKD now have another treatment option to help manage their anemia.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">A marketing authorisation application for daprodustat is currently under review with the European Medicines Agency, with a regulatory decision anticipated in the first half of 2023. In June 2020, daprodustat tablets were approved by Japan’s Ministry of Health, Labour and Welfare for the treatment of patients with anemia of CKD. In Japan, the brand name for daprodustat is <em style="box-sizing: inherit;">Duvroq</em>, where it is the market leader and preferred HIF-PHI.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;"><span style="box-sizing: inherit; font-weight: 600;">Tony Wood, President and Chief Scientific Officer, GSK, said: </span>“Over the last several decades, there has been little innovation in anemia of CKD. We are proud to have developed <em style="box-sizing: inherit;">Jesduvroq</em> as a new oral treatment where there is a patient desire for more options.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">The FDA approval is based on results from the ASCEND-D trial, assessing the efficacy and safety of <em style="box-sizing: inherit;">Jesduvroq</em> for the treatment of anemia of CKD in patients on dialysis. Results were published in the <em style="box-sizing: inherit;">New England Journal of Medicine</em> with additional results published in the <em style="box-sizing: inherit;">New England Journal of Medicine</em> supplementary appendix.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">The Safety Information for <em style="box-sizing: inherit;">Jesduvroq </em>includes a boxed warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, and thrombosis of vascular access. <em style="box-sizing: inherit;">Jesduvroq</em> increases the risk of thrombotic vascular events, including major adverse cardiovascular events (MACE). Targeting a hemoglobin level greater than 11 g/dL is expected to further increase the risk of death and arterial venous thrombotic events, as occurs with erythropoietin stimulating agents (ESAs), which also increase erythropoietin levels. No trial has identified a hemoglobin target level, dose of <em style="box-sizing: inherit;">Jesduvroq</em>, or dosing strategy that does not increase these risks. Use the lowest dose of <em style="box-sizing: inherit;">Jesduvroq</em> sufficient to reduce the need for red blood cell transfusions. <em style="box-sizing: inherit;">Jesduvroq</em> has not been shown to improve quality of life, fatigue, or patient well-being. <em style="box-sizing: inherit;">Jesduvroq</em> is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia or for treatment of anemia of chronic kidney disease in patients who are not on dialysis.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">CKD is an increasing global health burden affecting 700 million patients worldwide, with an estimated one in seven patients also developing anemia.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1,</span><span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2</span> When left untreated or undertreated, anemia of CKD is associated with poor clinical outcomes and leads to a substantial burden on patients and healthcare systems.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">3</span> There is an unmet need for oral treatment options with efficacy and safety comparable to current treatments.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;"><span style="box-sizing: inherit; font-weight: 600;">LaVarne Burton, President and Chief Executive Officer, American Kidney Fund, said: </span><em style="box-sizing: inherit;">“</em>Anemia of CKD can be a debilitating condition that is challenging to manage. This news means that patients on dialysis who are living with anemia of CKD now have another treatment option to help manage their anemia.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">A marketing authorisation application for daprodustat is currently under review with the European Medicines Agency, with a regulatory decision anticipated in the first half of 2023. In June 2020, daprodustat tablets were approved by Japan’s Ministry of Health, Labour and Welfare for the treatment of patients with anemia of CKD. In Japan, the brand name for daprodustat is <em style="box-sizing: inherit;">Duvroq</em>, where it is the market leader and preferred HIF-PHI.</span></p></div><div style="text-align: justify;"><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">The FDA approval is based on results from the ASCEND-D trial, assessing the efficacy and safety of <em style="box-sizing: inherit;">Jesduvroq</em> for the treatment of anemia of CKD in patients on dialysis. Results were published in the <em style="box-sizing: inherit;">New England Journal of Medicine</em> with additional results published in the <em style="box-sizing: inherit;">New England Journal of Medicine</em> supplementary appendix.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">The Safety Information for <em style="box-sizing: inherit;">Jesduvroq </em>includes a boxed warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, and thrombosis of vascular access. <em style="box-sizing: inherit;">Jesduvroq</em> increases the risk of thrombotic vascular events, including major adverse cardiovascular events (MACE). Targeting a hemoglobin level greater than 11 g/dL is expected to further increase the risk of death and arterial venous thrombotic events, as occurs with erythropoietin stimulating agents (ESAs), which also increase erythropoietin levels. No trial has identified a hemoglobin target level, dose of <em style="box-sizing: inherit;">Jesduvroq</em>, or dosing strategy that does not increase these risks. Use the lowest dose of <em style="box-sizing: inherit;">Jesduvroq</em> sufficient to reduce the need for red blood cell transfusions. <em style="box-sizing: inherit;">Jesduvroq</em> has not been shown to improve quality of life, fatigue, or patient well-being. <em style="box-sizing: inherit;">Jesduvroq</em> is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia or for treatment of anemia of chronic kidney disease in patients who are not on dialysis.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">CKD is an increasing global health burden affecting 700 million patients worldwide, with an estimated one in seven patients also developing anemia.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1,</span><span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2</span> When left untreated or undertreated, anemia of CKD is associated with poor clinical outcomes and leads to a substantial burden on patients and healthcare systems.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">3</span> There is an unmet need for oral treatment options with efficacy and safety comparable to current treatments.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;"><span style="box-sizing: inherit; font-weight: 600;">LaVarne Burton, President and Chief Executive Officer, American Kidney Fund, said: </span><em style="box-sizing: inherit;">“</em>Anemia of CKD can be a debilitating condition that is challenging to manage. This news means that patients on dialysis who are living with anemia of CKD now have another treatment option to help manage their anemia.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: inherit;">A marketing authorisation application for daprodustat is currently under review with the European Medicines Agency, with a regulatory decision anticipated in the first half of 2023. In June 2020, daprodustat tablets were approved by Japan’s Ministry of Health, Labour and Welfare for the treatment of patients with anemia of CKD. In Japan, the brand name for daprodustat is <em style="box-sizing: inherit;">Duvroq</em>, where it is the market leader and preferred HIF-PHI.</span></p><p style="background-color: white; box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="color: #202227;">https://en.wikipedia.org/wiki/Daprodustat</span></p><p style="background-color: white; box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="color: #202227;">https://go.drugbank.com/drugs/DB11682</span></p></div><div style="text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-jesduvroq-daprodustat-anemia-caused-chronic-kidney-adults-dialysis-5966.html" style="text-align: left;">FDA Approves Jesduvroq (daprodustat) for Anemia Caused by Chronic Kidney Disease for Adults on Dialysis</a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-47659787560750812612024-02-24T12:31:00.001+05:302024-02-24T12:31:00.120+05:30FDA Approves Miebo (perfluorohexyloctane) Ophthalmic Solution for the Treatment of the Signs and Symptoms of Dry Eye Disease<div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"><br /><br /></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEj8rT3QVTdYkk6ZHlHJiNoO4_1aeGUB7QnkDQo0yfmeUQaPWjxK99-iY096kE7JDL5F9bTobcLh9N2Y2FM-RLPfis8MsbkLtVqmjzXAYdXvmwrINRXmFu2qXt-YCw3p22FyPMr2nBsIBywj7GeScoLttZaxQzkvc3oS51eoIdunChvSfG-hqDVC9_fA" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="250" data-original-width="250" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEj8rT3QVTdYkk6ZHlHJiNoO4_1aeGUB7QnkDQo0yfmeUQaPWjxK99-iY096kE7JDL5F9bTobcLh9N2Y2FM-RLPfis8MsbkLtVqmjzXAYdXvmwrINRXmFu2qXt-YCw3p22FyPMr2nBsIBywj7GeScoLttZaxQzkvc3oS51eoIdunChvSfG-hqDVC9_fA" width="240" /></a></div><div class="separator" style="clear: both; text-align: center;"><br /></div><div class="separator" style="clear: both; text-align: justify;"><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Bausch + Lomb Corporation, announced that the U.S. Food and Drug Administration (FDA) has approved Miebo<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">™</span> (perfluorohexyloctane ophthalmic solution; formerly known as NOV03), for the treatment of the signs and symptoms of dry eye disease (DED). Miebo is the first and only FDA-approved treatment for DED that directly targets tear evaporation.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“Today’s FDA approval of Miebo further advances DED treatment by addressing a significant unmet need for millions of people suffering with this disease,” said Brent Saunders, chairman and CEO, Bausch + Lomb. “We are proud to bring to market the first and only prescription eye drop approved in the United States for the treatment of DED that directly targets evaporation. We expect to make Miebo commercially available in the second half of this year.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">DED affects millions of Americans and is one of the most common ocular surface disorders.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1</span> A leading cause of DED is excessive tear evaporation, which due to an altered tear lipid layer, is often associated with the clinical signs of Meibomian gland dysfunction (MGD). An unstable tear film triggers increased ocular surface desiccation, inflammation and damage to the ocular surface.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2,3 </span>Miebo is designed to reduce tear evaporation at the ocular surface.<span style="box-sizing: inherit; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">4,5</span></span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">In GOBI and MOJAVE, two phase 3 pivotal clinical trials which enrolled more than 1,200 patients (randomized 1:1 to Miebo or hypotonic saline) with a history of DED and clinical signs of MGD, Miebo consistently met its primary clinical sign and patient-reported symptom endpoint.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">"In the two pivotal clinical trials, Miebo addressed the persistent and chronic nature of DED by providing sustained improvement in both the signs and symptoms of DED,” said Preeya Gupta, M.D., cornea and cataract surgeon, Triangle Eye Consultants, Raleigh, North Carolina. “Because Miebo inhibits evaporation, it may be an appropriate treatment option for patients whose tear evaporation exceeds tear supply.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“Tear evaporation, which is a leading driver of DED, presents a significant treatment challenge. With the approval of Miebo, eye care professionals can now take a new approach to DED therapy with a first-in-class water- and preservative-free prescription treatment option that specifically addresses tear evaporation,” said Paul Karpecki, O.D., director, Cornea and External Disease, Kentucky Eye Institute, and associate professor, University of Pikeville, Kentucky College of Optometry.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="background-color: transparent; text-align: center;"><br /></span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="background-color: transparent; text-align: center;">Ref : <a href="FDA Approves Miebo (perfluorohexyloctane) Ophthalmic Solution for the Treatment of the Signs and Symptoms of Dry Eye Disease" target="_blank">Read More</a> </span></p></div></div><br /><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-68946611511599349892024-02-22T12:56:00.001+05:302024-02-22T12:56:00.135+05:30FDA Approves Lumryz (sodium oxybate) for Cataplexy or Excessive Daytime Sleepiness in Adults with Narcolepsy<div style="text-align: justify;"><br /></div><div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=sodium+oxybate" target="_blank">Lumryz</a></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEjF0a21kIHsMsCfyUtwkm8wBItInhw7den1jyZdxUhQm0WaKI7f3SmjlLIYIYqw9FGp_1uQqx72n5RukRE9DZQv1eeWA7o-4xOUWj4opDvkjXEinCtoQm0OHwjHR6DTFc6NAa0ebMlTp22ospKdmHBJSRf95CyIOP8L9gGw8Bw42g988RYxJW1oyPzP" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="300" data-original-width="300" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEjF0a21kIHsMsCfyUtwkm8wBItInhw7den1jyZdxUhQm0WaKI7f3SmjlLIYIYqw9FGp_1uQqx72n5RukRE9DZQv1eeWA7o-4xOUWj4opDvkjXEinCtoQm0OHwjHR6DTFc6NAa0ebMlTp22ospKdmHBJSRf95CyIOP8L9gGw8Bw42g988RYxJW1oyPzP" width="240" /></a></div><br /><br /><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><br /></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Avadel Pharmaceuticals plc a biopharmaceutical company announced the U.S. Food & Drug Administration (FDA) final approval to Lumryz, an extended-release formulation of sodium oxybate indicated to be taken once at bedtime for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. With final approval, Lumryz becomes the first and only FDA approved once-at-bedtime oxybate for people living with narcolepsy. Lumryz was additionally granted Orphan Drug Exclusivity by the FDA.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“Today’s landmark approval and receipt of Orphan Drug Exclusivity represents a major milestone for both Avadel and people living with narcolepsy. As we have heard from key stakeholders, previously approved narcolepsy therapies have the potential to disrupt sleep by either causing insomnia or through forced awakening during the middle of the night for their crucial second dose. Lumryz can now offer people with narcolepsy the opportunity for an uninterrupted night sleep while receiving the full benefit of their prescribed treatment in one single bedtime dose that addresses their symptoms of narcolepsy,” said Greg Divis, Chief Executive Officer of Avadel. “We would like to thank the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have tirelessly partnered with us throughout the drug development process and look forward to providing the narcolepsy community access to now approved Lumryz.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Narcolepsy is a chronic neurological condition that impairs the brain's ability to regulate the sleep-wake cycle. The condition affects approximately one in 2,000 people in the United States with the cardinal symptom of EDS. Additional symptoms can vary by person but may include disrupted nighttime sleep, a sudden loss of muscle tone usually triggered by strong emotion (cataplexy), sleep paralysis and hallucinations.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">“This long-awaited therapy for people living with narcolepsy fills a critical unmet need by avoiding the burden of a second middle-of-the-night dose that immediate-release oxybate products require. The once-at-bedtime dosing regimen of Lumryz may help restore a more natural sleep-wake cycle,” said Michael J. Thorpy, M.D., an investigator from the REST-ON Phase 3 trial and Director at the Sleep-Wake Disorders Center at Montefiore Medical Center and Professor of Neurology at the Albert Einstein College of Medicine.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The FDA’s final approval of Lumryz was based on positive results from the pivotal Phase 3 REST-ON clinical study completed in March 2020. In the REST-ON Phase 3 trial, once-at-bedtime Lumryz demonstrated highly statistically significant (p<0.001) and clinically meaningful improvement compared to placebo across all three co-primary endpoints (Maintenance of Wakefulness Test, Clinical Global Impression-Improvement and mean weekly cataplexy attacks) for all three doses evaluated, 6, 7.5 and 9 grams.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">With this approval, the FDA has also found Lumryz to be clinically superior to currently marketed twice-nightly oxybate products and granted Lumryz seven years of Orphan Drug Exclusivity. In particular, FDA found that Lumryz makes a major contribution to patient care over currently available, twice-nightly oxybate products by providing a once-nightly dosing regimen that avoids nocturnal arousal to take a second dose. The FDA's Orphan Drug program is designed to support the development of drugs that treat a condition affecting less than 200,000 U.S. patients. The seven-year market exclusivity for Lumryz began on the date of FDA approval, May 1, 2023.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“For people living with narcolepsy, and for all of us who advocate for this community, the approval of Lumryz is an important step forward,” said Julie Flygare, JD, President and CEO of Project Sleep. “People living with narcolepsy will finally have a new treatment option to manage EDS and cataplexy, and the fact that this new oxybate option allows for reduced dosing frequency is a game-changing advancement that shows Avadel’s commitment to understanding the patient experience. We look forward to continued collaboration with Avadel as part of a shared mission to positively impact the lives of people with narcolepsy.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Lumryz has a boxed warning as a central nervous system depressant, and for its potential for abuse and misuse. Lumryz is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the Lumryz REMS. Most common adverse reactions (incidence > 5% and greater than placebo) reported for all doses of Lumryz combined were nausea, dizziness, enuresis, headache, and vomiting.</p></div><div style="text-align: justify;">https://en.wikipedia.org/wiki/Sodium_oxybate</div><div><br /></div><div><br /></div><a href="https://www.drugs.com/newdrugs/fda-approves-lumryz-sodium-oxybate-cataplexy-excessive-daytime-sleepiness-adults-narcolepsy-6008.html">FDA Approves Lumryz (sodium oxybate) for Cataplexy or Excessive Daytime Sleepiness in Adults with Narcolepsy</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-83987759058588841042024-02-20T12:49:00.001+05:302024-02-20T12:49:00.125+05:30FDA Approves Mydcombi (tropicamide and phenylephrine hydrochloride) Ophthalmic Spray for Inducing Mydriasis<p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=Phenylephrine" target="_blank">phenylephrine hydrochloride</a></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEiPWbhAVOIvP6YpHA0squdTdt17wN9Hf3YLz52hsTF50ejlzNBZOgl7vgBAPsTk0L22NM6bjIaRCCKTuC_TNLWYie6Aouxmw5T13ce8UpP06V8Xc2OH49EwDmK39C30TGlXPPVKQ3h4-t21gpI_cwNhMQgcULaJW-ThuRrNLt6lS_45TsWquald2gwZ" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="220" data-original-width="401" height="176" src="https://blogger.googleusercontent.com/img/a/AVvXsEiPWbhAVOIvP6YpHA0squdTdt17wN9Hf3YLz52hsTF50ejlzNBZOgl7vgBAPsTk0L22NM6bjIaRCCKTuC_TNLWYie6Aouxmw5T13ce8UpP06V8Xc2OH49EwDmK39C30TGlXPPVKQ3h4-t21gpI_cwNhMQgcULaJW-ThuRrNLt6lS_45TsWquald2gwZ" width="320" /></a></div><div class="separator" style="clear: both; text-align: center;"><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;">Phenylephrine</b></div><div class="separator" style="clear: both; text-align: center;"><br /></div><div class="separator" style="clear: both; text-align: center;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEheOzCRTJNS1Iqd2oWgrXikWE2o4kyNJAUAM98OxYVTKX4uNcpIAfR5RygxmsY7cZsP2kU7PJ2G03un0pnhnyt-ko44wmMdok8NeESO47gnVdV81rDaGlRwYdixtmViS6LXepCve4BysGi5t1Y10g2gzm3upKzKL0K7Zu_Sj_HPDymt-OaBMSVVY6D9" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="249" data-original-width="398" height="200" src="https://blogger.googleusercontent.com/img/a/AVvXsEheOzCRTJNS1Iqd2oWgrXikWE2o4kyNJAUAM98OxYVTKX4uNcpIAfR5RygxmsY7cZsP2kU7PJ2G03un0pnhnyt-ko44wmMdok8NeESO47gnVdV81rDaGlRwYdixtmViS6LXepCve4BysGi5t1Y10g2gzm3upKzKL0K7Zu_Sj_HPDymt-OaBMSVVY6D9" width="320" /></a></div><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;">Tropicamide</b><br /><br /></div><br />Eyenovia, Inc, announced the U.S. Food and Drug Administration (FDA) approval of Mydcombi (tropicamide and phenylephrine hydrochloride ophthalmic spray) 1%/2.5% for inducing mydriasis for diagnostic procedures and in conditions where short term pupil dilation is desired. This represents the first approved fixed dose combination of tropicamide and phenylephrine in the United States and also the first product using Eyenovia’s proprietary Optejet device to be approved by any regulatory authority.<p></p><p style="background: white; margin-bottom: 15.0pt; margin-left: 0cm; margin-right: 0cm; margin-top: 9.0pt; text-align: justify;"><span style="color: #202227; font-family: "Segoe UI",sans-serif; font-size: 13.0pt;">Mydcombi is designed to
improve the efficiency of the estimated 106 million office-based comprehensive
eye exams performed every year in the United States, as well as the estimated 4
million pharmacologic mydriasis applications for cataract surgery. The product
is contraindicated and should not be used in patients with known
hypersensitivity to any component of the formulation.<o:p></o:p></span></p><p style="background: white; box-sizing: inherit; margin: 9pt 0cm 15pt; text-align: justify;"><span style="color: #202227; font-family: "Segoe UI",sans-serif; font-size: 13.0pt;">“The approval of Mydcombi, our first FDA approved product,
represents the culmination of years of tireless effort by the entire Eyenovia
team, and I would like to express my sincere gratitude to the associates and
technical experts who helped advance this important program through this
transformational milestone,” stated Michael Rowe, chief executive officer of
Eyenovia. “We look forward to introducing Mydcombi to key offices beginning
this summer while we bring our internal manufacturing capabilities on-line for
2024.”<o:p></o:p></span></p><p style="background: white; box-sizing: inherit; margin: 9pt 0cm 15pt; text-align: justify;"><span style="color: #202227; font-family: "Segoe UI",sans-serif; font-size: 13.0pt;">“Perhaps more importantly, FDA approval of Mydcombi provides
critical validation of the Optejet as it is the first product approved using
the Optejet platform, which is core not only to our internal development
programs, including MicroLine for presbyopia, but our partnered programs as
well. We see opportunities to unlock significant opportunities in the future
treatment of other ophthalmic conditions including glaucoma and dry eye. I am
confident in our ability to maintain our current momentum.”<o:p></o:p></span></p><p style="background: white; box-sizing: inherit; margin: 9pt 0cm 15pt; text-align: justify;"><span style="color: #202227; font-family: "Segoe UI",sans-serif; font-size: 13.0pt;">“I am proud of our team for this significant achievement – which
represents many ‘firsts’ for eye care,” stated Dr. Sean Ianchulev, Founder and
Chairman of Eyenovia’s Board of Directors. “The use of eye dropper bottles has
presented challenges for dosing in ophthalmologic settings in millions of
patients. We can do better now using sophisticated micro-array print delivery
with physiologic dosing that is similar to the natural tear film volume.”<o:p></o:p></span></p><p style="background: white; margin-bottom: 15.0pt; margin-left: 0cm; margin-right: 0cm; margin-top: 9.0pt; text-align: justify;"><span style="background-color: transparent; font-size: 17.3333px; text-align: left;"><span style="color: #202227; font-family: Segoe UI, sans-serif;">https://en.wikipedia.org/wiki/Phenylephrine</span></span></p><p style="background: white; margin: 9pt 0cm 15pt; text-align: left;"><span style="color: #202227; font-family: Segoe UI, sans-serif;"><span style="font-size: 17.3333px;">https://en.wikipedia.org/wiki/Tropicamide</span></span></p><p style="background: white; margin: 9pt 0cm 15pt; text-align: left;"><span style="color: #202227; font-family: Segoe UI, sans-serif;"><span style="font-size: 17.3333px;"><a href="FDA Approves Mydcombi (tropicamide and phenylephrine hydrochloride) Ophthalmic Spray for Inducing Mydriasis" target="_blank">Read More </a></span></span></p><div class="display-ad display-ad-injection display-ad-prebid display-ad-lazy display-ad-300 display-ad-300x250" data-google-query-id="CNGdndKqg_8CFTMe1QodGqMPQA" id="display-ad-injection-1" style="background-color: white; box-sizing: initial; clear: both; margin-bottom: 24px !important; margin-left: 0px; margin-right: 0px; margin-top: 24px !important; margin: 24px 0px; min-height: 262px; padding: 0px; width: 302px;"><span face="-apple-system, BlinkMacSystemFont, Segoe UI, Segoe UI Symbol, Helvetica Neue, Roboto, Arial, sans-serif"><span><div class="separator" style="clear: both; text-align: center;"><div class="separator" style="clear: both; color: #202227; font-size: 17px; text-align: center;"><br /></div></div></span></span></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-61080735930106778552024-02-15T09:03:00.001+05:302024-02-15T09:03:00.353+05:30FDA Grants Accelerated Approval for Qalsody (tofersen) for the Treatment of Amyotrophic Lateral Sclerosis Associated with a Mutation in the SOD1 Gene<div style="text-align: justify;"><br /></div><div style="text-align: justify;"><span style="background-color: white; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;">Biogen Inc. (Nasdaq: BIIB) announced the U.S. Food and Drug Administration (FDA) approval of Qalsody (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (</span><em style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;">SOD1</em><span style="background-color: white; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;">) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with Qalsody. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).</span><span style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1</span><span style="background-color: white; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;"> The ongoing Phase 3 ATLAS study of tofersen in people with presymptomatic </span><em style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;">SOD1</em><span style="background-color: white; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;">-ALS will serve as the confirmatory trial.</span></div><div style="text-align: justify;"><span style="background-color: white; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;"><br /></span></div><div style="text-align: justify;"><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Neurofilaments are proteins that are released from neurons when they are damaged, making them a marker of neurodegeneration.<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">6</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">“For more than a decade, Biogen has been steadfast in our commitment to pursuing treatments for ALS, and I want to thank the scientists as well as the entire ALS community who have all worked tirelessly to bring this first-of-its-kind treatment to people with <em style="box-sizing: inherit;">SOD1</em>-ALS,” said Christopher A. Viehbacher, President and Chief Executive Officer of Biogen. “Today also marks a pivotal moment in ALS research as we gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in <em style="box-sizing: inherit;">SOD1</em>-ALS. We believe this important scientific advancement will further accelerate innovative drug development for ALS.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Qalsody is the first approved treatment to target a genetic cause of ALS. Biogen collaborated with Ionis Pharmaceuticals on the early development of tofersen.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Warnings and precautions associated with Qalsody were serious neurologic events, including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis. If symptoms consistent with myelitis, radiculitis papilledema, elevated intracranial, or aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care. Management may require interruption or discontinuation of Qalsody. The most common adverse reactions that occurred in ≥10% of Qalsody treated participants and more than the placebo arm were pain, fatigue, arthralgia, cerebrospinal (CSF) white blood cell increased, and myalgia.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“Since <em style="box-sizing: inherit;">SOD1</em> mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments. Qalsody offers families who have lost generation after generation in the prime of their life to this devastating disease a therapy targeting the underlying cause of <em style="box-sizing: inherit;">SOD1</em>-ALS. Today marks an important moment in ALS research as Qalsody is the first ALS treatment approved based on a biomarker,” said Jean Swidler, chair of Genetic ALS & FTD: End the Legacy. “We are excited to see what future therapies are developed now that it is understood that lowering levels of neurofilament provides important evidence that a treatment is affecting the neurodegenerative process.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The efficacy of Qalsody was assessed in a 28-week randomized, double-blind, placebo-controlled clinical study in patients 23 to 78 years of age with weakness attributable to ALS and a <em style="box-sizing: inherit;">SOD1</em> mutation confirmed by a central laboratory. One hundred eight (108) patients were randomized 2:1 to receive treatment with either Qalsody 100 mg (n=72) or placebo (n=36) for 24 weeks (3 loading doses followed by 5 maintenance doses). Concomitant riluzole and/or edaravone use was permitted for patients and at baseline 62% of patients were taking riluzone, and 8% of patients were taking edaravone.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Over 28 weeks in VALOR, participants in the primary analysis population (n=60) treated with Qalsody experienced less decline from baseline as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) compared to placebo, though the results were not statistically significant (Qalsody-placebo adjusted mean difference [95% CI]: 1.2 [-3.2, 5.5]). In the overall intent-to-treat population (n=108), Qalsody-treated participants experienced a 55% reduction in plasma NfL compared to a 12% increase in placebo-treated participants (difference in geometric mean ratios for Qalsody to placebo: 60%; nominal p<0.0001). Additionally, levels of CSF SOD1 protein, an indirect measure of target engagement, were reduced by 35% in the Qalsody-treated group compared to 2% in the corresponding placebo group (difference in geometric mean ratios for Qalsody to placebo: 34%; nominal p<0.0001).</p></div><div style="text-align: justify;"><span style="background-color: white; font-size: 17px;"><span style="color: #202227; font-family: -apple-system, BlinkMacSystemFont, Segoe UI, Segoe UI Symbol, Helvetica Neue, Roboto, Arial, sans-serif;">https://en.wikipedia.org/wiki/Tofersen</span></span></div><div style="text-align: justify;"><span style="background-color: white; font-size: 17px;"><span style="color: #202227; font-family: -apple-system, BlinkMacSystemFont, Segoe UI, Segoe UI Symbol, Helvetica Neue, Roboto, Arial, sans-serif;">https://go.drugbank.com/drugs/DB14782</span></span></div><div style="text-align: justify;"><span style="background-color: white; font-size: 17px;"><span style="color: #202227; font-family: -apple-system, BlinkMacSystemFont, Segoe UI, Segoe UI Symbol, Helvetica Neue, Roboto, Arial, sans-serif;"><br /></span></span></div><div style="text-align: justify;"><span style="background-color: white; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px;"><br /></span></div><a href="https://www.drugs.com/newdrugs/fda-grants-accelerated-approval-qalsody-tofersen-amyotrophic-lateral-sclerosis-associated-mutation-6001.html">FDA Grants Accelerated Approval for Qalsody (tofersen) for the Treatment of Amyotrophic Lateral Sclerosis Associated with a Mutation in the SOD1 Gene</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-46058428378981336322024-02-14T08:56:00.001+05:302024-02-14T08:56:00.137+05:30FDA Approves Abilify Asimtufii (aripiprazole) for the Treatment of Schizophrenia or Maintenance Monotherapy Treatment of Bipolar I Disorder in Adults<div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=aripiprazole" target="_blank">aripiprazole</a></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Otsuka Pharmaceutical Co., Ltd. (Otsuka) and Lundbeck Pharmaceuticals LLC (Lundbeck) announced the U.S. Food and Drug Administration (FDA) approval of the New Drug Application (NDA) for Abilify Asimtufii<span style="box-sizing: inherit; font-weight: 600;">®</span> (aripiprazole) extended-release injectable suspension for intramuscular use, a once-every-two-months injection for the treatment of schizophrenia in adults or for maintenance monotherapy treatment of bipolar I disorder in adults.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Abilify Asimtufii offers two months of sustained therapeutic concentrations with one dose. Each dose is provided in a single-chamber, prefilled syringe, and is administered by a healthcare professional to appropriate patients via intramuscular injection in the gluteal muscle. Long-acting injectables provide continuous delivery of antipsychotic medication and can maintain therapeutic plasma concentrations, which may help to maintain symptom control of schizophrenia and bipolar I disorder.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>"We are pleased to offer this new treatment option for people living with schizophrenia or bipolar I disorder that may delay the time to relapse," said John Kraus, M.D., Ph.D., executive vice president and chief medical officer at Otsuka Pharmaceutical Development & Commercialization, Inc. "This approval underscores Otsuka's commitment to innovate and continuously evolve to meet the needs of the communities we serve."</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The efficacy of Abilify Asimtufii is based on the adequate and well-controlled studies of Abilify Maintena (aripiprazole) in the treatment of schizophrenia or maintenance treatment of bipolar I disorder in adults. The aripiprazole concentrations of Abilify Asimtufii were explored in a pharmacokinetic bridging study which was a 32-week, open-label, multiple-dose, randomized, parallel-arm, multicenter study (N=266) in patients living with schizophrenia and bipolar I disorder. The once-every-two-months, long-acting injectable formulation in 960 mg and 720 mg prefilled syringes delivers sustained plasma concentrations similar to that demonstrated in studies with aripiprazole monohydrate once-monthly, long-acting injectable, resulting in similar sustained efficacy.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>"This approval is important news for patients, families, and healthcare providers. We hope that the use of Abilify Asimtufii in treatment plans will have a positive impact on those living with schizophrenia or bipolar I disorder," said Johan Luthman, executive vice president, R&D, Lundbeck. "We are grateful to the patients and researchers who made this major milestone possible."</blockquote><p></p></div><div><br /></div><div>https://en.wikipedia.org/wiki/Aripiprazole</div><div><br /></div><div><br /></div><a href="https://www.drugs.com/newdrugs/fda-approves-abilify-asimtufii-aripiprazole-schizophrenia-maintenance-monotherapy-bipolar-disorder-6004.html">FDA Approves Abilify Asimtufii (aripiprazole) for the Treatment of Schizophrenia or Maintenance Monotherapy Treatment of Bipolar I Disorder in Adults</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-81818207404671920792024-02-13T13:03:00.001+05:302024-02-13T13:03:00.126+05:30FDA Approves Uzedy (risperidone) Extended-Release Injectable Suspension for the Treatment of Schizophrenia in Adults<div><br /></div><div><span style="font-family: Playfair Display;">In continuation of my update on <span style="background-color: white; font-weight: 600;"><a href="https://www.med-chemist.com/search?q=risperidone" target="_blank">Uzedy (risperidone)</a></span></span></div><div><span style="font-family: Playfair Display;"><br /></span></div><div><span style="font-family: Playfair Display;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEghxCov92_UtC-ZfBdpjZoHkfCsCGO6iwVx8gseMc1q0s9oiBoRzEGaDUSHFphuoKlfe-RYkzqUkoWOww6PO499TK4ZcHyeW5yvn6iFF802ptgJQpWol4Z4D4cLwUxfgWn3GbrMNRGmfOzJB0uWPi-DjKJq4Vmfm3AEuS7-B0yqCF41v6dOVaCEAAhx" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="300" data-original-width="300" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEghxCov92_UtC-ZfBdpjZoHkfCsCGO6iwVx8gseMc1q0s9oiBoRzEGaDUSHFphuoKlfe-RYkzqUkoWOww6PO499TK4ZcHyeW5yvn6iFF802ptgJQpWol4Z4D4cLwUxfgWn3GbrMNRGmfOzJB0uWPi-DjKJq4Vmfm3AEuS7-B0yqCF41v6dOVaCEAAhx" width="240" /></a></div><br /></span><p style="background-color: white; box-sizing: inherit; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Teva Pharmaceuticals, announced the U.S. Food and Drug Administration (FDA) approval of Uzedy (risperidone) extended-release injectable suspension for the treatment of schizophrenia in adults. Uzedy is the first subcutaneous, long-acting formulation of risperidone that utilizes SteadyTeq™, a copolymer technology proprietary to MedinCell that controls the steady release of risperidone. Therapeutic blood concentrations are reached within 6-24 hours of a single dose. </span></p><p style="background-color: white; box-sizing: inherit; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;"></span></p><blockquote><span style="font-family: Playfair Display;">“Uzedy embodies Teva’s commitment to bringing innovative advances to patients and to providing people living with schizophrenia an important new treatment option that was designed to address certain treatment challenges and may decrease the risk of relapse,” said Richard Francis, President and CEO of Teva. “The approval of Uzedy is a culmination of a multidisciplinary effort across Teva and MedinCell to bring this important treatment to market. This milestone is a testament to advancing our robust biopharmaceutical pipeline of innovative medicines that aim to support more people living with mental health disorders and neurological diseases in the coming years.”</span></blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;">Approximately 80% of patients with schizophrenia experience multiple relapses over the first five years of treatment,<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2</span> most commonly due to suboptimal adherence to treatment with oral antipsychotics. Each relapse carries a biological risk of loss of function, treatment refractoriness, and changes in brain morphology.<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">3,4</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;">Schizophrenia is a chronic, progressive and severely debilitating mental health disorder that affects how one thinks, feels and acts.<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">5</span> This approval is based on data from two Phase 3 trials evaluating Uzedy in patients with schizophrenia: TV46000-CNS-30072 (the RISE Study – The Risperidone Subcutaneous Extended-Release Study) and TV46000-CNS-30078 (the SHINE Study – A Study to Test TV-46000 for Maintenance Treatment of Schizophrenia).</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"></p><blockquote style="text-align: justify;">“The approval of the first product formulated with our technology is a pivotal moment for MedinCell and for the many patients who will benefit,” said Christophe Douat, CEO of MedinCell. “We are committed to supporting patients through innovative therapy options. It continues to be a wonderful journey with Teva, an ideal partner to harness the full potential of Uzedy. Our technology reaching commercial stage marks the start of an exciting new era for MedinCell and we are extremely proud to share this very special moment with all our employees and shareholders.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; margin: 12px 0px 20px; padding: 0px;"><span style="font-size: 17px;">The use of novel SteadyTeq technology in Uzedy controls the release of risperidone over time. The initiation of treatment requires no loading dose or oral supplementation. Therapeutic blood concentrations are reached within 6-24 hours of a single dose.</span><span style="font-size: 12px;"> </span></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;">“Treatments for schizophrenia are largely prescribed as daily oral medications, which can present challenges with adherence due to missed doses. Lack of adherence to treatment with oral antipsychotics is the most common cause of relapse in schizophrenia so there’s a role for therapies that are dosed in one- or two-month dosing intervals to help prevent relapse,” said Christoph Correll, MD, professor of psychiatry at the Zucker School of Medicine, Hempstead, NY. “As a clinician, I am excited to now have a new treatment option that reduces the risk of relapse<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1</span> for this complex disease and helps address some of the barriers around receiving schizophrenia treatment.”</p><div class="display-ad display-ad-injection display-ad-prebid display-ad-lazy display-ad-300 display-ad-300x250" data-google-query-id="CMGjwpKwg_8CFRpNaAodHKoC7A" id="display-ad-injection-1" style="background-color: white; box-sizing: initial; clear: both; margin-bottom: 24px !important; margin-left: 0px; margin-right: 0px; margin-top: 24px !important; min-height: 262px; padding: 0px; text-align: justify; width: 302px;"><span style="color: #202227; font-family: -apple-system, BlinkMacSystemFont, Segoe UI, Segoe UI Symbol, Helvetica Neue, Roboto, Arial, sans-serif;"><span style="font-size: 17px;">https://pubchem.ncbi.nlm.nih.gov/compound/Risperidone#section=2D-Structure</span></span></div></div><div class="display-ad display-ad-injection display-ad-prebid display-ad-lazy display-ad-300 display-ad-300x250" data-google-query-id="CMGjwpKwg_8CFRpNaAodHKoC7A" id="display-ad-injection-1" style="background-color: white; box-sizing: initial; clear: both; margin-bottom: 24px !important; margin-left: 0px; margin-right: 0px; margin-top: 24px !important; min-height: 262px; padding: 0px; text-align: justify; width: 302px;"><span style="color: #202227; font-family: -apple-system, BlinkMacSystemFont, Segoe UI, Segoe UI Symbol, Helvetica Neue, Roboto, Arial, sans-serif;"><span style="font-size: 17px;"><br /></span></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></div><div style="text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-uzedy-risperidone-extended-release-injectable-suspension-schizophrenia-adults-6006.html"><span style="color: black; font-family: Playfair Display;">FDA Approves Uzedy (risperidone) Extended-Release Injectable Suspension for the Treatment of Schizophrenia in Adults</span></a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-42297309438137436642024-02-07T14:06:00.001+05:302024-02-07T14:06:00.164+05:30FDA Grants Accelerated Approval to Filspari (sparsentan) for the Reduction of Proteinuria in IgA Nephropathy<div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;">Travere Therapeutics, Inc. (Nasdaq: TVTX) announced the U.S. Food and Drug Administration (FDA) approval to </span>Filspari<span style="background-color: white; color: #202227;">™ (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g.</span></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;"><br /></span></span></div><div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEiKzmllj0nsYXo9eAV-Y2SxgcJqlKMGiJ_ar4tBzij7GUIj1TXwOA_h7-a1tPo1issbbNd_o_b_Iv__pfHGabo1L6y40Bdd8UjHl0MNEgalLk6c_F5IjE8MEohyI2yelZip5gfeZKLj0CNXdgAtqB7YA0iME85svTmrjfiFqw7-lF_dCReI9bErCo2L" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="761" data-original-width="800" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEiKzmllj0nsYXo9eAV-Y2SxgcJqlKMGiJ_ar4tBzij7GUIj1TXwOA_h7-a1tPo1issbbNd_o_b_Iv__pfHGabo1L6y40Bdd8UjHl0MNEgalLk6c_F5IjE8MEohyI2yelZip5gfeZKLj0CNXdgAtqB7YA0iME85svTmrjfiFqw7-lF_dCReI9bErCo2L" width="252" /></a></div><br /><br /></span></span></div><div><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"><span style="font-family: Playfair Display;">This indication is granted under accelerated approval based on reduction in proteinuria. It has not been established whether Filspari slows kidney function decline in patients with IgAN. The continued approval of Filspari may be contingent upon confirmation of a clinical benefit in the ongoing Phase 3 PROTECT Study, which is designed to demonstrate whether Filspari slows kidney function decline. Topline results from the two-year confirmatory endpoints in the PROTECT Study are expected in the fourth quarter of 2023 and are intended to support traditional approval of Filspari.</span></p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"><span style="font-family: Playfair Display;">Filspari, a once-daily oral medication is designed to selectively target two critical pathways in the disease progression of IgAN (endothelin-1 and angiotensin II), and is the first and only non-immunosuppressive therapy approved for the treatment of this condition. IgAN is a rare kidney disease (RKD) and a leading cause of kidney failure due to glomerular disease, affecting up to 150,000 people in the U.S., with approximately 30,000 to 50,000 of such patients estimated to be addressable under the indication approved via accelerated approval. The Company expects Filspari to be available beginning the week of February 27, 2023, and will be providing a comprehensive patient support program throughout the patient’s treatment journey.</span></p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"></p><blockquote><span style="font-family: Playfair Display;">“The accelerated approval of Filspari is a significant milestone on our path to advancing a transformative treatment for the IgA nephropathy community,” said Eric Dube, Ph.D., president and chief executive officer, Travere Therapeutics. “As a first-of-its-kind, non-immunosuppressive therapy, we believe Filspari has the potential to ultimately become the new standard of care for IgA nephropathy and offer hope to those living with this condition who until now have had few treatment options. We are grateful to the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have worked alongside us to develop this innovative first-in-class therapy.”</span></blockquote><p></p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"></p><blockquote style="text-align: justify;"><span style="font-family: Playfair Display;">“Today’s approval of Filspari sets the stage for a new standard of care for IgA nephropathy patients. A high proportion of individuals diagnosed with this disease do not sufficiently respond to the historical standard treatment, which has been therapies that are not indicated for IgA nephropathy. These treatments include hypertension drugs such as angiotensin-receptor blockers (ARBs), angiotensin-converting enzyme (ACE) inhibitors and systemic glucocorticoids. As a result, many patients have struggled to manage their disease and have progressed more quickly to kidney failure,” said Dr. Brad Rovin, MD, Medical Director at Ohio State University Center for Clinical Research Management, Director of the Division for Nephrology, and steering committee member for the PROTECT clinical trial. “The approval of this innovative treatment is founded in data from the largest head-to-head Phase 3 clinical trial in IgA nephropathy. It is exciting to see that adult patients who are at risk of rapid disease progression, many of whom have waited a very long time for a treatment like this, now have hope for a better future.”</span></blockquote><p></p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"><span style="font-family: Playfair Display;">The approval of Filspari, granted under the FDA’s accelerated approval pathway, is based on clinically meaningful and statistically significant improvements in proteinuria compared to an active comparator in the pivotal and ongoing Phase 3 PROTECT Study, the largest head-to-head interventional study to date in IgAN. The PROTECT Study is a global, randomized, multicenter, double-blind, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of Filspari, compared to 300 mg of irbesartan, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite maximal tolerated ACE or ARB therapy. </span></p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"><span style="font-family: Playfair Display;">https://en.wikipedia.org/wiki/Sparsentan</span></p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"><a href="https://www.drugs.com/newdrugs/fda-grants-accelerated-approval-filspari-sparsentan-reduction-proteinuria-iga-nephropathy-5976.html" style="background-color: transparent; outline-width: 0px !important; text-align: left; user-select: auto !important;"><span style="font-family: Playfair Display;">FDA Grants Accelerated Approval to Filspari(sparsentan) for the Reduction of Proteinuria in IgA Nephropathy</span></a></p></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-31627467504607721352024-02-03T10:18:00.003+05:302024-02-03T10:18:00.132+05:30FDA Approves Veozah (fezolinetant) for the Treatment of Vasomotor Symptoms Due to Menopause<div><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Astellas Pharma Inc. announced the U.S. Food and Drug Administration (FDA) approval of Veozah (fezolinetant) 45 mg once daily for the treatment of moderate to severe vasomotor symptoms (VMS) due to menopause on May 12. Veozah is the first nonhormonal neurokinin 3 (NK3) receptor antagonist approved to treat VMS due to menopause.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;"></span></p><div class="separator" style="clear: both; text-align: center;"><span style="font-family: Playfair Display;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEhxL9sGRrNR-D07eyqBVMTyaGCs1GCPYXnM5zBwSFX3rJ6F3pf2R287abdZ_jjk3B8-mQMwnwtGWGlrfZv7qhA3w-cjCJ1GuwQEWYND9aVgaTNibic3nLzI2b4kidbR6S17o6PC99wPm5c4cPEqnHMno3nMJPQwpqR-iIZ3rLMhWkI4azPLZodAZRSX" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="538" data-original-width="800" height="215" src="https://blogger.googleusercontent.com/img/a/AVvXsEhxL9sGRrNR-D07eyqBVMTyaGCs1GCPYXnM5zBwSFX3rJ6F3pf2R287abdZ_jjk3B8-mQMwnwtGWGlrfZv7qhA3w-cjCJ1GuwQEWYND9aVgaTNibic3nLzI2b4kidbR6S17o6PC99wPm5c4cPEqnHMno3nMJPQwpqR-iIZ3rLMhWkI4azPLZodAZRSX" width="320" /></a></span></div><span style="font-family: Playfair Display;"><br /><br /></span><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“Today’s approval of fezolinetant is a significant and, I believe, long awaited milestone for individuals in the U.S. who experience moderate to severe vasomotor symptoms during the menopausal transition,” said Genevieve Neal-Perry, M.D., Ph.D., Chair, UNC School of Medicine Department of Obstetrics and Gynecology. “This therapy is based on our understanding of the biology behind hot flashes. I’m excited to know that patients will have the option to choose this nonhormonal treatment.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: center;"><span style="font-family: Playfair Display;">Before menopause, there is a balance between estrogens (hormones made by a woman’s ovaries) and neurokinin B (NKB), a brain chemical. This balance regulates the body’s temperature control center located in a specific area of the brain. As the body goes through menopause, estrogens decline and this balance is disrupted. This imbalance can lead to very uncomfortable symptoms called VMS. Veozah helps to restore the balance by blocking NKB in the temperature control center to reduce the number and intensity of hot flashes.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px;"><span style="font-family: Playfair Display;"></span></p><blockquote><blockquote style="text-align: center;"><span style="font-family: Playfair Display;">“Veozah uses a novel mechanism of action to target the root cause of VMS due to menopause,” said Marci English, Vice President and Head of BioPharma Development, Astellas. “FDA approval of this new treatment for moderate to severe VMS due to menopause is a testament to Astellas’ commitment to delivering innovative therapies in areas of unmet need that have been underserved, including women’s health.”</span></blockquote></blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px;"><span style="font-family: Playfair Display;">The approval is supported by results from the BRIGHT SKY™ program, which included three Phase 3 clinical trials as part of a development program that collectively enrolled over 3,000 individuals across the U.S., Canada and Europe. Results from the SKYLIGHT 1™ and SKYLIGHT 2™ pivotal trials characterize the efficacy and safety of fezolinetant for the treatment of moderate to severe VMS due to menopause. Data from the SKYLIGHT 4™ safety study further characterizes the long-term safety profile of fezolinetant.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;"><br /></span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">VMS, characterized by hot flashes and/or night sweats, are common symptoms of menopause. VMS are the most common symptoms of menopause for which women seek treatment. In the U.S., about 60% to 80% of women experience these symptoms during or after the menopausal transition. VMS can have a disruptive impact on women’s daily activities and overall quality of life.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“Today’s approval of fezolinetant is a significant and, I believe, long awaited milestone for individuals in the U.S. who experience moderate to severe vasomotor symptoms during the menopausal transition,” said Genevieve Neal-Perry, M.D., Ph.D., Chair, UNC School of Medicine Department of Obstetrics and Gynecology. “This therapy is based on our understanding of the biology behind hot flashes. I’m excited to know that patients will have the option to choose this nonhormonal treatment.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Before menopause, there is a balance between estrogens (hormones made by a woman’s ovaries) and neurokinin B (NKB), a brain chemical. This balance regulates the body’s temperature control center located in a specific area of the brain. As the body goes through menopause, estrogens decline and this balance is disrupted. This imbalance can lead to very uncomfortable symptoms called VMS. Veozah helps to restore the balance by blocking NKB in the temperature control center to reduce the number and intensity of hot flashes.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">“Veozah uses a novel mechanism of action to target the root cause of VMS due to menopause,” said Marci English, Vice President and Head of BioPharma Development, Astellas. “FDA approval of this new treatment for moderate to severe VMS due to menopause is a testament to Astellas’ commitment to delivering innovative therapies in areas of unmet need that have been underserved, including women’s health.”</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">The approval is supported by results from the BRIGHT SKY™ program, which included three Phase 3 clinical trials as part of a development program that collectively enrolled over 3,000 individuals across the U.S., Canada and Europe. Results from the SKYLIGHT 1™ and SKYLIGHT 2™ pivotal trials characterize the efficacy and safety of fezolinetant for the treatment of moderate to severe VMS due to menopause. Data from the SKYLIGHT 4™ safety study further characterizes the long-term safety profile of fezolinetant.</span></p><p style="background-color: white; box-sizing: inherit; margin: 12px 0px 20px; padding: 0px;"><span style="color: #202227; font-family: Playfair Display;">Ref : </span><span style="background-color: transparent;"><span style="color: #202227; font-family: Playfair Display;">https://en.wikipedia.org/wiki/Fezolinetant</span></span></p><div class="display-ad display-ad-injection display-ad-prebid display-ad-lazy display-ad-300 display-ad-300x250" data-google-query-id="CKbD0YnIgP8CFY2XZgIdrp8KCQ" id="display-ad-injection-1" style="background-color: white; box-sizing: initial; clear: both; color: #202227; margin-bottom: 24px !important; margin-left: 0px; margin-right: 0px; margin-top: 24px !important; min-height: 262px; padding: 0px; width: 302px;">F<a href="https://www.drugs.com/newdrugs/fda-approves-veozah-fezolinetant-vasomotor-due-menopause-6013.html" style="background-color: transparent;"><span style="font-family: Playfair Display;">DA Approves Veozah (fezolinetant) for the Treatment of Vasomotor Symptoms Due to Men</span>opause</a></div></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-1699438691456886102024-01-25T12:49:00.001+05:302024-01-25T12:49:22.674+05:30FDA Approves RizaFilm (rizatriptan) Oral Film for the Treatment of Acute Migraine<div><br /></div><div><br /></div><div><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEgxrIB21P20FpZw_goxBEsh2YcmQxtNGHMucFBjR9dsouIfyBgHReTgpjbhC4IEFixYJMn-W3zcBTC_K8vVwOqr1GAqETpGvKptWcFAJpA953RQZ09uopul2GeG76VEFbXyUlsw3NYZQvdZahVcSsG2CGo3VCE9kuWLOH6LYw1ZJlQ7w2esRSySgFiu" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="300" data-original-width="300" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEgxrIB21P20FpZw_goxBEsh2YcmQxtNGHMucFBjR9dsouIfyBgHReTgpjbhC4IEFixYJMn-W3zcBTC_K8vVwOqr1GAqETpGvKptWcFAJpA953RQZ09uopul2GeG76VEFbXyUlsw3NYZQvdZahVcSsG2CGo3VCE9kuWLOH6LYw1ZJlQ7w2esRSySgFiu" width="240" /></a></div><div class="separator" style="clear: both; text-align: justify;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=rizatriptan" target="_blank">rizatriptan</a></div><br /><br /></div><div><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;">IntelGenx Corp. (TSX:IGX)(OTCQB:IGXT), announced the U.S. Food and Drug Administration approval of RizaFilm<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span> VersaFilm<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span> 505(b)(2) new drug application (NDA) for the treatment of acute migraine.</p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;">RizaFilm® is a proprietary oral thin film formulation of rizatriptan benzoate, the active ingredient in Merck & Co.'s Maxalt<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span>. The global migraine drugs market was valued at nearly $3 billion in 2021 and is expected to reach nearly $11 billion by 2030, representing a compound annual growth rate of 15.6%.</p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;">In December 2018, IntelGenx entered into a definitive licensing, development and supply agreement with Gensco<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span> Pharma (“Gensco”) for the exclusive commercialization of RizaFilm<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span> in the United States. Under the terms of the agreement, IntelGenx is entitled to receive royalty payments based on net profits of RizaFilm<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span>; and is eligible to receive pre-specified payments upon the achievement of certain regulatory and commercial milestones.</p><p align="justify" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"></p><blockquote>“Following a successful pre-approval inspection by the FDA of our Montreal manufacturing facility earlier this month, we are thrilled to reach this milestone and excited to soon introduce what will be the first oral thin film for the treatment of acute migraines available in the U.S.,” said Andre Godin, IntelGenx’s President and CFO. “According to the American Migraine Foundation, 39 million or 12% of Americans suffer from migraine, which is the second leading cause of disability nationwide. We are looking forward to working with our commercialization partner, Gensco, to bring this innovative migraine therapeutic to patients seeking convenient administration and quick relief from their pain. In addition to these benefits, RizaFilm<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span> is well suited to the approximately 80% of patients who have migraine-related nausea<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">3</span>, as well as those who have difficulty swallowing.”</blockquote><p></p><div class="display-ad display-ad-injection display-ad-prebid display-ad-lazy display-ad-300 display-ad-300x250" data-google-query-id="CKLoyr_GiP8CFVOKaAodfwkDFQ" id="display-ad-injection-1" style="background-color: white; box-sizing: initial; clear: both; margin-bottom: 24px !important; margin-left: 0px; margin-right: 0px; margin-top: 24px !important; margin: 24px 0px; min-height: 262px; padding: 0px; width: 302px;"><span face="-apple-system, BlinkMacSystemFont, Segoe UI, Segoe UI Symbol, Helvetica Neue, Roboto, Arial, sans-serif" style="color: #202227;"><span style="font-size: 17px;"><a href="https://www.drugs.com/newdrugs/fda-approves-rizafilm-rizatriptan-oral-film-acute-migraine-5997.html" target="_blank">Rea More..</a> </span></span></div><div class="display-ad display-ad-injection display-ad-prebid display-ad-lazy display-ad-300 display-ad-300x250" data-google-query-id="CKLoyr_GiP8CFVOKaAodfwkDFQ" id="display-ad-injection-1" style="background-color: white; box-sizing: initial; clear: both; margin-bottom: 24px !important; margin-left: 0px; margin-right: 0px; margin-top: 24px !important; margin: 24px 0px; min-height: 262px; padding: 0px; width: 302px;"><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="color: #202227; font-size: 17px;">https://en.wikipedia.org/wiki/Rizatriptan</span></div></div><div><br /></div><a href="https://www.drugs.com/newdrugs/fda-approves-rizafilm-rizatriptan-oral-film-acute-migraine-5997.html">FDA Approves RizaFilm (rizatriptan) Oral Film for the Treatment of Acute Migraine</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-26845984163716141382024-01-06T14:17:00.006+05:302024-01-30T10:07:13.382+05:30FDA Approves Joenja (leniolisib) for the Treatment of Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)<div style="text-align: justify;"><span style="font-family: Playfair Display;"><span style="background-color: white; color: #202227;">Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announced the USFDA approval of </span>Joenja<span style="background-color: white; color: #202227;"> (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja® application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja® is expected to launch in the US in early April and </span><span style="background-color: white; color: #202227;">Dr. Eveline Wu, MD, MSCR, Division Chief, Paediatric Rheumatology & Associate Professor of Paediatric Rheumatology and Allergy/Immunology at The University of North Carolina School of Medicine, said:</span></span></div><div style="text-align: justify;"><span style="background-color: white; color: #202227;"><span style="font-family: Playfair Display;"><br /></span></span></div><div style="text-align: justify;"><span style="background-color: white; color: #202227;"><span style="font-family: Playfair Display;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEhY4n2k-02MeKPcyyPVN-7i9vkxsLyCFBvyiBECyaObhLNK2TOzyAvfXEcROFslw459adateK1piBtK_Knqve7BVFacLLrhBiJT_YNugaUK_WfuUf3z8ElxPdjI6rX1RXd7a2sl1KiERSl-M0Q7lUanY3__M3-s27ZjKJGOyL_hnAb_3Fr64oq7mbyc" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="429" data-original-width="1024" height="134" src="https://blogger.googleusercontent.com/img/a/AVvXsEhY4n2k-02MeKPcyyPVN-7i9vkxsLyCFBvyiBECyaObhLNK2TOzyAvfXEcROFslw459adateK1piBtK_Knqve7BVFacLLrhBiJT_YNugaUK_WfuUf3z8ElxPdjI6rX1RXd7a2sl1KiERSl-M0Q7lUanY3__M3-s27ZjKJGOyL_hnAb_3Fr64oq7mbyc" width="320" /></a></div><br /><br /></span></span></div><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote><span style="font-family: Playfair Display;">"The FDA approval of Joenja® is an exciting moment for the APDS community and offers to transform the treatment pathway for patients and families affected by this rare disease. This approval means that they will, for the first time, have access to an approved treatment, which has the potential to change the standard of care for the patient population suffering from APDS."</span></blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Vicki Modell, co-founder of the Jeffrey Modell Foundation, an international, non-profit, organization dedicated to helping individuals and family members affected by primary immunodeficiency disorders, commented:</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote><span style="font-family: Playfair Display;">"The approval of Pharming's Joenja® is an important step toward making a difference in the lives of individuals living with APDS who experience severe, life-altering and progressive symptoms. The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community. The Jeffrey Modell Foundation's mission of hope, advocacy and action is dedicated to early diagnosis, genetic sequencing, treatments and ultimately, future cures for primary immunodeficiencies."</span></blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Sijmen de Vries, Chief Executive Officer of Pharming, commented:</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">"This FDA approval of Joenja® is an important milestone for people living with APDS who will now have access to the first approved treatment option specifically for this debilitating disease. Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS. We are grateful to the patients, caregivers, and physicians who participated in the clinical trials who have made today's approval a reality. I would also like to thank the Pharming and the Novartis teams who have supported the development of Joenja® and can, therefore, be justifiably proud of this FDA approval.</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">Today also marks a landmark event for Pharming and demonstrates our commitment to transforming the lives of patients who suffer from rare diseases. The approval and near-term launch of Joenja®, our second commercial product, brings us closer to our goal of becoming a leading global rare disease company dedicated to patient communities with unmet medical needs."</span></p><p style="background-color: white; box-sizing: inherit; color: #202227; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="font-family: Playfair Display;">APDS is a rare primary immunodeficiency that was first characterized in 2013 and is currently estimated to affect 1 to 2 people per million. It is caused by genetic variants in either one of two identified genes, known as <em style="box-sizing: inherit;">PIK3CD</em> or <em style="box-sizing: inherit;">PIK3R1</em>, which are vital to the normal development and function of immune cells in the body. While people with APDS may suffer from a wide variety of symptoms, the most common are frequent and severe infections of the ears, sinuses, and upper and lower respiratory tracts. Infections usually begin in infancy. People with APDS are susceptible to swollen lymph nodes or an enlarged spleen (splenomegaly), as well as autoimmunity and inflammatory symptoms. People with APDS may also be at higher risk for cancers like lymphoma.</span></p><div style="text-align: justify;"><span style="font-family: Playfair Display;">https://en.wikipedia.org/wiki/Leniolisib</span></div><div><span style="font-family: Playfair Display;"><br /></span></div><div><a href="https://www.drugs.com/newdrugs/fda-approves-joenja-leniolisib-activated-phosphoinositide-3-kinase-delta-syndrome-apds-5991.html"><span style="font-family: Playfair Display;">FDA Approves Joenja (leniolisib) for the Treatment of Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)</span></a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-24000977519672348882023-01-23T06:00:00.008+05:302023-01-23T06:00:00.172+05:30FDA Approves Iyuzeh (latanoprost ophthalmic solution) for the Reduction of Elevated Intraocular Pressure (IOP) in Patients with Open-Angle Glaucoma or Ocular Hypertension<div><p class="p3" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span class="s3" style="box-sizing: inherit;">In continuation of my update on </span><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: left;"><a href="https://www.med-chemist.com/search?q=Latanoprost" target="_blank">Latanoprost</a></b></p><p class="p3" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEjMKkaLY2w0fx7hyEjA52NKk3xRFAKhGv_EUEhB3HunR1HfCron8XxtJh5WX22MhVNfAd-qto2SuZYb-IIwmlAdkuREYh4gUwr_JJbi_HroUapx7Y5uBpv1ttCOgN3gxaTqfL41ce7FybZCVRf4ieaJ8KpVwhXJye71nbK_jTcI7UTfICBwBr8P11W-" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="216" data-original-width="330" height="209" src="https://blogger.googleusercontent.com/img/a/AVvXsEjMKkaLY2w0fx7hyEjA52NKk3xRFAKhGv_EUEhB3HunR1HfCron8XxtJh5WX22MhVNfAd-qto2SuZYb-IIwmlAdkuREYh4gUwr_JJbi_HroUapx7Y5uBpv1ttCOgN3gxaTqfL41ce7FybZCVRf4ieaJ8KpVwhXJye71nbK_jTcI7UTfICBwBr8P11W-" width="320" /></a></div><br /><p style="text-align: justify;"><span class="s3" style="box-sizing: inherit;">Thea Pharma, Inc.</span> (“Thea”), the U.S. subsidiary of Europe’s leading independent pharmaceutical company, <span class="s3" style="box-sizing: inherit;">Laboratoires Théa</span>, dedicated to the research, development and commercialization of ophthalmic products, announced U.S. Food and Drug Administration (FDA) approval of the New Drug Application (NDA) of Iyuzeh™ (latanoprost ophthalmic solution) 0.005% for the reduction of elevated intraocular pressure (IOP) in patients with open-angle glaucoma (OAG) or ocular hypertension (OHT).</p><p></p><p class="p3" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Iyuzeh™ is the first and only clinically proven formulation of latanoprost available in the United States that is preservative-free. Iyuzeh™ is formulated without any of the preservatives commonly used in topical ocular preparations, including benzalkonium chloride (BAK). Iyuzeh™ has demonstrated consistent IOP-lowering effects and proven tolerability across multiple trials in the U.S. and Europe. In randomized, controlled clinical trials of patients with OAG or OHT with mean baseline IOP of 19-24 mmHg, Iyuzeh™ lowered IOP by 3 – 8 mmHg versus 4 – 8 mmHg by XALATAN<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span> (latanoprost ophthalmic solution) 0.005%, which is preserved with BAK.</p><p class="p3" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“The approval of Iyuzeh™ is a significant milestone for Théa Group, as this is our first FDA approval for a prescription ophthalmic medicine, for our U.S. subsidiary,” said Jean-Frédéric Chibret, President of the Théa Group. “Marketed outside of the U.S. as Monoprost<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">®</span>, the market leading prostaglandin analogue (PGA) in volume, is available in over 46 countries around the world, including France, Germany, Spain, United Kingdom, Italy, and Canada. We are extremely proud to bring our unique preservative-free latanoprost eye drop, Iyuzeh™ to the U.S.</blockquote><p></p><p class="p3" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><p style="text-align: justify;">“Our novel patent protected formulation has been made possible by Théa’s innovative scientists. They were able to solve the challenges of solubilizing and stabilizing latanoprost such that Iyuzeh does not need to be manufactured, distributed, or stored at refrigerated temperatures unlike some other competitive brand and generic latanoprost and PGA products. Additionally, Théa is responding to an important unmet need across all stakeholders in the treatment of OAG and OHT. Many patients on preserved glaucoma medications experience moderate to severe signs and symptoms of ocular surface disease (OSD) that can cause discomfort for patients, frustration for physicians, and drive additional costs for payers,” said Susan Benton, Thea’s U.S. President. “We look forward to introducing Iyuzeh™ to U.S. eyecare practitioners in the second half of 2023.”</p><p></p><p class="p3" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="box-sizing: inherit; font-weight: 600;">About Iyuzeh™</span></p><p class="p3" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Iyuzeh™ (latanoprost ophthalmic solution) 0.005%, an opalescent, white to slightly yellow ophthalmic solution, is a topical formulation of latanoprost that is indicated for the reduction of elevated intraocular pressure (IOP) in patients with open-angle glaucoma (OAG) or ocular hypertension (OHT). Iyuzeh™ does not contain a preservative – it is the first and only preservative-free formulation of latanoprost, the most prescribed prostaglandin F2α analogue (PGA) in the United States. The recommended dosage of Iyuzeh™ is one drop in the affected eye(s) once daily in the evening. If one dose is missed, treatment should continue with the next dose as normal. Reduction of the IOP starts approximately 3 to 4 hours after administration and the maximum effect is reached after 8 to 12 hours. IOP reduction is present for at least 24 hours.</p><p class="p3" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In the two clinical trials conducted with Iyuzeh™ (latanoprost ophthalmic solution) 0.005%, the most frequently reported ocular adverse reactions were conjunctival hyperemia (34%) and eye irritation (19%) compared to XALATAN, the preserved 0.005% latanoprost reference product which reported conjunctival hyperemia (37%) and eye irritation (31%).</p></div><div style="text-align: justify;"><br /></div><div style="text-align: justify;">https://en.wikipedia.org/wiki/Latanoprost</div><span><a name='more'></a></span><div style="text-align: justify;"><br /></div><div style="text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-iyuzeh-latanoprost-ophthalmic-solution-reduction-elevated-intraocular-pressure-iop-5941.html">FDA Approves Iyuzeh (latanoprost ophthalmic solution) for the Reduction of Elevated Intraocular Pressure (IOP) in Patients with Open-Angle Glaucoma or Ocular Hypertension</a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-28483094123907876232023-01-20T06:00:00.004+05:302023-01-20T06:00:00.170+05:30FDA Approves Rezlidhia (olutasidenib) for Relapsed or Refractory Acute Myeloid Leukemia with a Susceptible IDH1 Mutation<div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Rigel Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) approval of Rezlidhia (olutasidenib) capsules for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. Rezlidhia is an oral, small molecule, inhibitor of mutated IDH1 designed to bind to and inhibit mIDH1 to reduce 2-hydroxyglutarate levels and restore normal cellular differentiation of myeloid cells.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEi2T6nI_1EIKe1tGo-Kj0oMjCKQ-j8HpbD6QUDHZwkp7RcSlJnYyYtxWPrwtOsQxRvOunjrxTeYby2x8N-V4GUt7G8YDQtjBJdCRhhO30ZvLcsasRftHTIsu3NH2z-fSFgKBJsdAMe3-p8N8IZLYp6VmCprf_NbeHrlFE9uk2S7vf5uw4xUd6n-J3xo" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="161" data-original-width="330" height="156" src="https://blogger.googleusercontent.com/img/a/AVvXsEi2T6nI_1EIKe1tGo-Kj0oMjCKQ-j8HpbD6QUDHZwkp7RcSlJnYyYtxWPrwtOsQxRvOunjrxTeYby2x8N-V4GUt7G8YDQtjBJdCRhhO30ZvLcsasRftHTIsu3NH2z-fSFgKBJsdAMe3-p8N8IZLYp6VmCprf_NbeHrlFE9uk2S7vf5uw4xUd6n-J3xo" width="320" /></a></div><br /><br /><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>"Rezlidhia is a novel, non-intensive monotherapy treatment in the relapsed/refractory AML setting demonstrating a CR+CRh rate of 35% in patients with over 90% of those responders in complete remission. The 25.9 months median duration of CR+CRh is a clinically meaningful improvement for AML patients and appears to be longer than currently available treatment options," said Jorge E. Cortes, M.D., Director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, and Phase 2 trial investigator. "Given the limited treatment options for adult patients with mIDH1 R/R AML, who typically have a poor prognosis, Rezlidhia may provide an effective, new treatment option with a well characterized safety profile."</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The FDA approval was supported by data from the open-label Phase 2 registrational study evaluating Rezlidhia monotherapy at a dose of 150 mg twice daily in 153 mIDH1 R/R AML patients. The efficacy-evaluable population was 147 patients who initiated Rezlidhia at least six months prior to the interim analysis cutoff date of June 18, 2021, and who had a centrally confirmed IDH1 mutation. The primary endpoint was a composite of a complete remission (CR) plus a complete remission with partial hematological recovery (CRh). CRh is defined as less than 5% blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets >50,000/microliter and absolute neutrophil count >500/microliter).</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Results from the trial demonstrated a 35% (51/147) CR+CRh rate in mIDH1 R/R AML patients, with a median duration of response of 25.9 months. The median time to CR or CRh was 1.9 months. Of the patients who achieved the primary endpoint of CR+CRh, 92% (47/51) were CR with a median duration of response of 28.1 months. Rezlidhia was well tolerated in the study with an adverse event profile largely characteristic of symptoms or conditions experienced by patients with AML undergoing treatment. Differentiation syndrome was observed in 16% of patients and was manageable in most cases with dose interruption and corticosteroids. Hepatotoxicity, presenting as increases in liver function parameters, occurred in 23% of patients and most cases were manageable with dose modifications.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>"We are delighted by the approval of Rezlidhia based on the strength of data supporting the efficacy and safety of the product," said Raul Rodriguez, Rigel's president and CEO. "Rezlidhia provides a new and important, oral therapy option for patients who typically have a poor clinical outcome. Additionally, this approval greatly strengthens and expands Rigel's commercial hematology-oncology portfolio. I would like to extend our sincerest thanks to all the patients, their families and caregivers, the doctors, the FDA, and our team members who have all contributed to the approval of Rezlidhia."</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In August 2022, Rigel and Forma Therapeutics, Inc. announced they entered an exclusive, worldwide license agreement to develop, manufacture and commercialize Rezlidhia. Under the terms of the agreement, Rigel will be responsible for the launch and commercialization of Rezlidhia in the U.S., and intends to work with potential partners to further develop and commercialize the product outside the U.S.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><span style="background-color: transparent;">REf : https://en.wikipedia.org/wiki/Olutasidenib</span></p></div><div style="text-align: justify;"></div><span><a name='more'></a></span><div style="text-align: justify;"><br /></div><div style="text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-rezlidhia-olutasidenib-relapsed-refractory-acute-myeloid-leukemia-susceptible-idh1-5935.html">FDA Approves Rezlidhia (olutasidenib) for Relapsed or Refractory Acute Myeloid Leukemia with a Susceptible IDH1 Mutation</a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-53961501965552999092023-01-19T06:00:00.004+05:302023-01-19T06:00:00.158+05:30FDA Approves Krazati (adagrasib) for Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) with a KRASG12C Mutation<div><u style="box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; text-align: justify;">Mirati Therapeutics, Inc.</u><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;"> </span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;">(NASDAQ: MRTX), a targeted oncology company, announced the U.S. Food and Drug Administration (FDA) approval of </span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;">Krazati</span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;">™ (adagrasib), a targeted treatment option for adult patients with KRAS</span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="box-sizing: inherit; color: #202227; font-size: 0.75rem; line-height: 0; position: relative; text-align: justify; top: -0.5em; vertical-align: baseline;">G12C</span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;">-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.</span></div><div><div class="module_related-document" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 0px; padding: 0px; text-align: justify;"><br /></div><div class="module_related-document" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 0px; padding: 0px; text-align: justify;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEj-UMkn5rXhfnFfo1kvOVehO-ABUCaDrbCsrC19QhryBUolEfY7-IRyQ66ZwDavGmYYIbmok-cTmIxeWu34cuP7mf6HbbXRyfVepbRekPM-12GyjjuIuoJwqjZCM-DbqXr7NVqIcGYAJzDrQmvW2fYDhDFqZQ9x9PwMeJwbbwneNG2f0ixtJGVlS_T9" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="334" data-original-width="330" height="240" src="https://blogger.googleusercontent.com/img/a/AVvXsEj-UMkn5rXhfnFfo1kvOVehO-ABUCaDrbCsrC19QhryBUolEfY7-IRyQ66ZwDavGmYYIbmok-cTmIxeWu34cuP7mf6HbbXRyfVepbRekPM-12GyjjuIuoJwqjZCM-DbqXr7NVqIcGYAJzDrQmvW2fYDhDFqZQ9x9PwMeJwbbwneNG2f0ixtJGVlS_T9" width="237" /></a></div></div><div class="module_body clearfix" style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 0px; padding: 0px;"><div class="xn-content" style="box-sizing: inherit; margin: 0px; padding: 0px;"><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">This indication is approved under accelerated approval based on objective response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of a clinical benefit in a confirmatory trial(s).</p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">"The FDA approval of Krazati is a positive development for thousands of patients with KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span> mutations, including the approximately 14% of patients with NSCLC adenocarcinomas histology that harbor a KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span> mutation.<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1</span> Mirati is thrilled to make Krazati available in a tablet formulation to patients in the U.S. with advanced NSCLC who have progressed beyond a first-line treatment for the historically difficult-to-treat KRAS mutation," <u style="box-sizing: inherit;">David Meek</u>, chief executive officer, Mirati Therapeutics, Inc., continued, "We look forward to continuing to advance our Krazati development program including several monotherapy and combination studies in KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span>-mutated solid tumors."</p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Krazati has demonstrated a positive benefit-risk profile with accelerated approval based on the Phase 2 registration-enabling cohort of the KRYSTAL-1 study, evaluating Krazati 600 mg capsules administered orally twice daily in 116 patients with KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span>-mutated advanced NSCLC who previously received treatment with a platinum-based regimen and an immune checkpoint inhibitor. The primary efficacy endpoints were confirmed ORR and DOR as evaluated by blinded independent central review (BICR) according to response evaluation criteria in solid tumors (RECIST v1.1).</p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The trial demonstrated an ORR of 43% (95% CI: 34-53) with 80% (95% CI: 71-87) of patients achieving disease control. The median DOR was 8.5 months (95% CI: 6.2-13.8).</p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In a pooled efficacy analysis (n=132) including Phase 1/1b NSCLC and registrational Phase 2 NSCLC cohorts from the KRYSTAL-1 study evaluating adagrasib as a single agent at 600 mg capsules orally twice daily, adagrasib showed an ORR of 44% and a disease control rate of 81% based on BICR, a median DOR of 12.5 months (95% CI, 7.3-NE) and median overall survival of 14.1 months (94% CI, 9.2-19.2).</p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The safety profile of Krazati was evaluated in a pooled patient population with NSCLC and other solid tumors as a single agent at 600 mg orally twice daily in 366 patients enrolled in KRYSTAL-1 and KRYSTAL-12. The most common (≥ 25%) adverse reactions were nausea, diarrhea, vomiting, fatigue, musculoskeletal pain, hepatotoxicity, renal impairment, edema, dyspnea and decreased appetite. Permanent discontinuation of Krazati due to an adverse reaction occurred in 13% of patients.</p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Although KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span> is the most common KRAS mutation in NSCLC, patients have had limited options for the treatment of this debilitating and difficult-to-treat condition.<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">2,3</span></p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">"The approval of Krazati offers an effective therapy for patients with advanced NSCLC harboring the KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span> mutation. The positive ORR and DOR results, as observed in previously treated patients with NSCLC harboring the KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span> mutation, demonstrate the effectiveness of Krazati as an option for these difficult-to-treat patients," said Shirish M. Gadgeel, MD, chief of the Division of Hematology and Oncology, Department of Internal Medicine, Henry Ford Cancer Institute/Henry Ford Health System.</p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>"KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span> in NSCLC is an area of high unmet need and new treatment options offer patients and our community new hope for survivorship," said Bonnie J. Addario, co-founder and board chair of the GO2 Foundation for Lung Cancer. "I'm pleased that patients have options, there's more awareness of this disease and we are all focused on improving the journeys of people living with KRAS<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">G12C</span>-mutated NSCLC."</blockquote><p></p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The Company partnered with Agilent and QIAGEN to develop blood- and tissue-based companion diagnostics (CDx), respectively, for Krazati that are now available. With tissue and blood modalities for companion diagnostics, patients have more flexibility, and clinicians have greater options for biomarker testing. These solutions help to personalize a patient's treatment path.</p><p style="box-sizing: inherit; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Mirati Therapeutics is launching Mirati & Me, a comprehensive program dedicated to supporting patients, caregivers and the oncology community including coverage and access, financial, educational and emotional support services. </p></div></div></div><div style="text-align: justify;"><br /></div><div style="text-align: justify;">Ref : https://en.wikipedia.org/wiki/Adagrasib</div><div style="text-align: justify;"><br /></div><span><a name='more'></a></span><div style="text-align: justify;"><br /></div><div style="text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-krazati-adagrasib-locally-advanced-metastatic-non-small-cell-lung-cancer-nsclc-5937.html">FDA Approves Krazati (adagrasib) for Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) with a KRASG12C Mutation</a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-68904421170275676062023-01-18T06:00:00.005+05:302023-01-18T06:00:00.167+05:30FDA Approves Olpruva (sodium phenylbutyrate) for Patients with Urea Cycle Disorders<div><br /></div><div style="text-align: justify;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=phenylbutyrate" target="_blank">Phenyl butyrate</a> </div><div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Acer Therapeutics Inc. and its collaboration partner, RELIEF THERAPEUTICS Holding SA (“Relief”), announced the U.S. Food and Drug Administration (FDA) approval Olpruva™ (sodium phenylbutyrate) of oral suspension in the U.S. for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: justify;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEjCyvSUyM4ZBPRKGFxwnxye3E8KsW4mFP4-YSawS8YcyC21XBePl368CXj3wu0KPtD1FhbrXOlz8pcKIQrg8jt4F5B3HN6boONSsUDylv93w_qTSMSjqDk4folrQD15hr52slw0909RwxbiqyK1X9LuBs1-xv0nOJzVZDWiFwZYWxHeI2-WOGnh4_ff" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="112" data-original-width="330" height="109" src="https://blogger.googleusercontent.com/img/a/AVvXsEjCyvSUyM4ZBPRKGFxwnxye3E8KsW4mFP4-YSawS8YcyC21XBePl368CXj3wu0KPtD1FhbrXOlz8pcKIQrg8jt4F5B3HN6boONSsUDylv93w_qTSMSjqDk4folrQD15hr52slw0909RwxbiqyK1X9LuBs1-xv0nOJzVZDWiFwZYWxHeI2-WOGnh4_ff" width="320" /></a></div><div style="text-align: justify;"><br /></div><div style="text-align: justify;"><br /></div><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote style="text-align: justify;">“The FDA’s approval of Olpruva, an innovative formulation of sodium phenylbutyrate packaged for the first time in single-dose envelopes, marks the culmination of our ongoing dedication to develop new and differentiated treatment options for those affected by rare diseases,” said Chris Schelling, chief executive officer and founder of Acer. “Patients who are living with UCDs now have an alternative treatment option with Olpruva™, to address some of the challenges they may have with existing therapy. We are pleased to be able to provide a new, approved treatment choice for those living with this challenging disease.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Mr. Schelling continued, “This approval represents the first FDA-approved product for Acer, validating our ability to identify and develop treatments where science can be applied in novel ways and make them available to patients as quickly and efficiently as possible. In addition, this approval unlocks our Marathon debt funding option and provides us with resources to advance our pipeline of investigational product candidates.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote style="text-align: justify;">“The daily challenges of living with a UCD can be overwhelming and emotionally draining for patients and their families,” said Tresa Warner, president of the National Urea Cycle Disorders Foundation. “We welcome new treatment options that can help patients, caregivers and their healthcare teams manage UCDs.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Olpruva’s™ approval triggers the availability of a $42.5 million term loan to Acer under the previously announced March 2022 loan agreement the Company entered into with affiliates of Marathon Asset Management L.P. If Acer requests and receives the loan proceeds, management believes it will have sufficient resources to fund current operations into H2 2023.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Olpruva™ has been approved as an oral suspension by the FDA for the treatment of patients with UCDs. UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments, if ammonia levels are not controlled.<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1</span> Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote style="text-align: justify;">“This FDA approval is a significant milestone for patients with UCDs in the U.S., offering an additional choice to manage their condition,” added Jack Weinstein, chief executive officer of Relief. “We look forward to building on Olpruva™’s approval in the U.S. and expanding its availability into other territories outside of the U.S.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Olpruva™ received FDA approval under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act (FDCA), a regulatory pathway that allows applicants to rely, at least in part, on third party data for approval. In its New Drug Application (NDA), Acer cited preclinical and clinical safety and efficacy data from the reference listed drug (RLD), BUPHENYL® powder, which is approved as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of CPS, OTC or AS. In its NDA, Acer also provided additional data including studies that evaluated the bioavailability and bioequivalence of Olpruva™ compared to BUPHENYL® powder. The data from these studies, presented at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting in April 2022 and the Genetic Metabolic Dieticians International (GMDI) Conference in May 2022, showed that Olpruva™ was bioequivalent to BUPHENYL® powder.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px;"></p><div style="text-align: justify;"><span style="font-weight: 600;">Commitment to Patient Access</span></div><div style="text-align: justify;">Acer intends to offer Navigator by Acer Therapeutics, a suite of integrated patient support services designed to facilitate access to therapy. Navigator by Acer Therapeutics is designed to assist UCD patients with support, access, education, and adherence.</div><p></p></div><div style="text-align: justify;">Ref : https://en.wikipedia.org/wiki/Sodium_phenylbutyrate</div><div style="text-align: justify;"><br /></div><div style="text-align: justify;"></div><span><a name='more'></a></span><div style="text-align: justify;"><br /></div><div style="text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-olpruva-sodium-phenylbutyrate-patients-urea-cycle-disorders-5953.html">FDA Approves Olpruva (sodium phenylbutyrate) for Patients with Urea Cycle Disorders</a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-87145674755094734522023-01-17T06:00:00.003+05:302023-01-17T06:00:00.174+05:30FDA Approves Sezaby (phenobarbital sodium powder for injection) for the Treatment of Neonatal Seizures<div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In continuation of my update on <a href="https://www.med-chemist.com/search?q=phenobarbital+" target="_blank">Phenobarbitol </a></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><br /></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEgs_JJspVVbnXxLq3_vsuKAc7KG_U0TMgghR15rKFCE1SAx2MCVbmu4fJ8c7t8CarOsGL6zrDMFqn6EMKe8Nab_FXN6zIqCeNUwBfkIZ0qsdKykUlff_Evxlx_SWRpET2NvEkpSX-EDRXRTup6hxdutVsEh2BoNneqs5CzxZGar_gULYEycb5BM9hU0" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="263" data-original-width="180" height="165" src="https://blogger.googleusercontent.com/img/a/AVvXsEgs_JJspVVbnXxLq3_vsuKAc7KG_U0TMgghR15rKFCE1SAx2MCVbmu4fJ8c7t8CarOsGL6zrDMFqn6EMKe8Nab_FXN6zIqCeNUwBfkIZ0qsdKykUlff_Evxlx_SWRpET2NvEkpSX-EDRXRTup6hxdutVsEh2BoNneqs5CzxZGar_gULYEycb5BM9hU0=w164-h165" width="164" /></a></div><br /><br /><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Sun Pharmaceutical Industries Limited and Sun Pharma Advanced Research Company Ltd. announced the U.S. Food and Drug Administration (US FDA) approval of Sezaby™ (phenobarbital sodium powder for injection) for the treatment of neonatal seizures.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">With this approval, Sezaby becomes the first and only product specifically indicated in the U.S. for the treatment of neonatal seizures in term and preterm infants. Sezaby is expected to be available in the U.S. in Q4FY23.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Sezaby is a benzyl alcohol-free and propylene glycol-free formulation of phenobarbital sodium powder for injection. It was granted orphan drug designation by the US FDA for the treatment of neonatal seizures.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Sezaby was recently licensed by SPARC to Sun Pharma. Under the terms of the license agreement, SPARC is eligible to receive a milestone payment on approval of Sezaby by the US FDA.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">“Sezaby is an exciting addition to our growing portfolio of specialty branded products in the U.S.,” said Abhay Gandhi, CEO North America, Sun Pharma. “As the first and only product specifically indicated to treat seizures in term and preterm infants, Sezaby has the potential to make a difference in the lives of patients and their families.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">“For years, physicians have had limited treatment options to manage neonates with seizures. SPARC is proud to have developed benzyl alcohol-free and propylene glycol-free phenobarbital sodium powder for injection as the first treatment option now approved by the US FDA,” said Anil Raghavan, CEO, SPARC. Sezaby was approved based on the results of NEOLEV2, a phase 2 study that evaluated levetiracetam compared to phenobarbital in the first-line treatment of neonatal seizures.</p></div><div style="text-align: justify;">Ref : https://en.wikipedia.org/wiki/Phenobarbital</div><div style="text-align: justify;"><br /></div><span><a name='more'></a></span><div style="text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-sezaby-phenobarbital-sodium-powder-neonatal-seizures-5932.html">FDA Approves Sezaby (phenobarbital sodium powder for injection) for the Treatment of Neonatal Seizures</a></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-77903476481935804452023-01-16T06:00:00.003+05:302023-01-16T06:00:00.170+05:30FDA Approves Airsupra (albuterol/budesonide) Metered-Dose Inhaler to Reduce the Risk of Asthma Exacerbations<div><br /></div><div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">In continuation of my update on <span style="font-weight: 600; text-align: left;"><a href="https://www.med-chemist.com/search?q=albuterol" target="_blank">albuterol</a>/<a href="https://www.med-chemist.com/search?q=+budesonide" target="_blank">budesonide</a></span></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEirxeOWGugwgrH3wKfVqgTXLrHlU6G3GjLwlyx-QvgVOmSytIY57l3Aatqmf8p-sJJDCFNdunEBGLs3Qg4PptiEaX365ZvRhEs-sHbYF06MGycGOCY4OP9G486-vkG0R8kQ_7BU37eMV7CFBuQHfGxYw6remeVqGqIomaxW_z3l9ltIwE7xD2vo8_P_" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="134" data-original-width="300" height="143" src="https://blogger.googleusercontent.com/img/a/AVvXsEirxeOWGugwgrH3wKfVqgTXLrHlU6G3GjLwlyx-QvgVOmSytIY57l3Aatqmf8p-sJJDCFNdunEBGLs3Qg4PptiEaX365ZvRhEs-sHbYF06MGycGOCY4OP9G486-vkG0R8kQ_7BU37eMV7CFBuQHfGxYw6remeVqGqIomaxW_z3l9ltIwE7xD2vo8_P_" width="320" /></a></div><div class="separator" style="clear: both; text-align: center;"><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;">Salbutamol</b><span style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;">,/</span><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;">albuterol</b></div><div class="separator" style="clear: both; text-align: center;"><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;"><br /></b></div><div class="separator" style="clear: both; text-align: center;"><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;"><br /></b></div><div class="separator" style="clear: both; text-align: center;"><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;"><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEh5HngD_GrdnpnJaH_QPDZZA9VD0lagpr1BOED5XERd0MtywkfDQrlgf6B7nfKHRfmHv3aB_Pso4a4D_lDxKw-5c77xYLVJnHeJnmc_4VKv81Va0hOkf4pVcqHjJlk3TiDgrQnsxIgfW1HH2PZTFjM19qPpbXAKyH86Fg_QmxhUnCh3icvz7QQEKzMY" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="231" data-original-width="375" height="197" src="https://blogger.googleusercontent.com/img/a/AVvXsEh5HngD_GrdnpnJaH_QPDZZA9VD0lagpr1BOED5XERd0MtywkfDQrlgf6B7nfKHRfmHv3aB_Pso4a4D_lDxKw-5c77xYLVJnHeJnmc_4VKv81Va0hOkf4pVcqHjJlk3TiDgrQnsxIgfW1HH2PZTFjM19qPpbXAKyH86Fg_QmxhUnCh3icvz7QQEKzMY" width="320" /></a></div><br /></b><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;">Budesonide//</b><b style="color: #202122; font-family: sans-serif; font-size: 14px; text-align: start;">Pulmicort</b></div><br /><br /><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Airsupra (albuterol/budesonide), formerly known as PT027, has been approved in the US for the as-needed treatment or prevention of bronchoconstriction and to reduce the risk of exacerbations in people with asthma aged 18 years and older.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The approval by the Food and Drug Administration (FDA) was based on results from the MANDALA and DENALI Phase III trials. In MANDALA, Airsupra significantly reduced the risk of severe exacerbations compared to albuterol in patients with moderate to severe asthma when used as an as-needed rescue medication in response to symptoms. Importantly, in the secondary endpoint of mean annualised total systemic corticosteroid exposure, Airsupra demonstrated a significant reduction compared to albuterol at the approved dose of 180mcg albuterol/160mcg budesonide. In DENALI, Airsupra significantly improved lung function compared to the individual components albuterol and budesonide in patients with mild to moderate asthma.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Airsupra is a first-in-class, pressurised metered-dose inhaler (pMDI), fixed-dose combination rescue medication containing albuterol, a short-acting beta2-agonist (SABA), and budesonide, an anti-inflammatory inhaled corticosteroid (ICS) in the US. It is being developed by AstraZeneca and Avillion.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Bradley E. Chipps, Past President of the American College of Allergy, Asthma & Immunology and Medical Director of Capital Allergy & Respiratory Disease Center in Sacramento, US, said: “People with asthma are at risk of severe exacerbations regardless of their disease severity or level of control. Current albuterol rescue inhalers alleviate acute symptoms, but do not treat the underlying inflammation in asthma. The approval of Airsupra means that for the first time, adults with asthma in the US have a rescue treatment to manage both their symptoms and the inflammatory nature of their disease.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “With patients experiencing more than 10 million asthma exacerbations each year in the US and uncontrolled asthma expected to cost the US economy billions of dollars in direct medical costs alone over the next 20 years, today’s positive decision is good news for those adults with asthma who make up more than 80% of asthma patients in the US. Physicians will be able to offer their patients Airsupra, an important new rescue treatment that reduces the risk of asthma exacerbations.”</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Asthma is a chronic, inflammatory respiratory disease with variable symptoms that affects as many as 262 million people worldwide.<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;"> </span>In the US over 21 million adults have asthma, representing more than 80% of the total number of people with asthma. Adults have 8.5 million exacerbations each year in the US. Uncontrolled asthma will cost the US economy an estimated $300 billion (in 2018 dollar values) in the next 20 years in direct medical costs alone.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The safety and tolerability of Airsupra in both trials were consistent with the known profiles of the components,<span style="box-sizing: inherit; font-size: 0.75rem; line-height: 0; position: relative; top: -0.5em; vertical-align: baseline;">1,2 </span>with the most common adverse events including headache, oral candidiasis, cough and dysphonia.</p></div><div style="text-align: justify;">https://en.wikipedia.org/wiki/Salbutamol</div><div style="text-align: justify;">https://en.wikipedia.org/wiki/Budesonide</div><span><a name='more'></a></span><div style="text-align: justify;"><br /></div><div style="text-align: justify;"><br /></div><a href="https://www.drugs.com/newdrugs/fda-approves-airsupra-albuterol-budesonide-metered-inhaler-reduce-risk-asthma-exacerbations-5956.html">FDA Approves Airsupra (albuterol/budesonide) Metered-Dose Inhaler to Reduce the Risk of Asthma Exacerbations</a><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0tag:blogger.com,1999:blog-6520560265126950473.post-65304797124011848972023-01-13T06:00:00.004+05:302023-01-13T06:00:00.172+05:30FDA Approves Sunlenca (lenacapavir) Twice-Yearly Treatment for People Living With Multi-Drug Resistant HIV<div style="text-align: left;"><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;">Gilead Sciences, Inc. (Nasdaq: GILD) announced that </span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;">Sunlenca</span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="box-sizing: inherit; color: #202227; font-size: 0.75rem; line-height: 0; position: relative; text-align: justify; top: -0.5em; vertical-align: baseline;">®</span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;"> </span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;">(lenacapavir), in combination with other antiretroviral(s) (ARV), has been granted approval by the U.S. Food and Drug Administration (FDA) for the treatment of HIV-1 infection in heavily treatment-experienced (HTE) adults with multi-drug resistant (MDR) HIV-1 infection. Sunlenca has a multi-stage mechanism of action distinguishable from other currently approved classes of antiviral agents and no known cross resistance exhibited</span><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;"> </span><em style="box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; text-align: justify;">in vitro </em><span face="-apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif" style="background-color: white; color: #202227; font-size: 17px; text-align: justify;">to other existing drug classes. Sunlenca offers a new, twice-yearly treatment option for adults with HIV that is not adequately controlled by their current treatment regimen.</span></div><div><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/a/AVvXsEheQBwop8CLxIOzRkMF3ZyGL8BQR4-tqRmiT0cwGpvzDn3FKCDZtNs8IS2D1ephcaRLde9X4yrH-YVKJpuXUd51fozgkjuDKgS6iNDHFUWjyRJ23l1fH1-NDTnbGQbzCwZbCVNCxjE1LUu4XuqLbxKc1HBDhs_9tFko4xMl1cCZ7s09D6eNUr3VkcGx" style="margin-left: 1em; margin-right: 1em;"><img alt="" data-original-height="192" data-original-width="262" height="235" src="https://blogger.googleusercontent.com/img/a/AVvXsEheQBwop8CLxIOzRkMF3ZyGL8BQR4-tqRmiT0cwGpvzDn3FKCDZtNs8IS2D1ephcaRLde9X4yrH-YVKJpuXUd51fozgkjuDKgS6iNDHFUWjyRJ23l1fH1-NDTnbGQbzCwZbCVNCxjE1LUu4XuqLbxKc1HBDhs_9tFko4xMl1cCZ7s09D6eNUr3VkcGx" width="320" /></a></div><br /><p></p><blockquote style="text-align: justify;">“An effective antiretroviral regimen can be devised for most people living with the virus; however, some people living with HIV no longer have durable viral suppression due to resistance to multiple classes of antiretroviral therapies,” said Sorana Segal-Maurer, MD, Director of the Dr. James J. Rahal Jr. Division of Infectious Diseases at NewYork-Presbyterian Queens, Professor of Clinical Medicine at Weill Cornell Medicine and the Site Principal Investigator for the CAPELLA trial. “The availability of new classes of antiretroviral drugs is critical for heavily treatment-experienced people with multi-drug resistant HIV. Following today’s decision from the FDA, lenacapavir helps to fill a critical unmet need for people with complex prior treatment histories and offers physicians a long-awaited twice-yearly option for these patients who otherwise have limited therapy choices."</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Despite the significant advances in ARV therapy, there remain numerous critical and pressing unmet needs for people living with HIV. This is particularly true for individuals who are heavily treatment experienced – which account for an estimated 2% of adults living with HIV who are on treatment globally –- and are unable to maintain virologic suppression due to resistance, intolerance or safety considerations. This type of complexity further increases the chance of treatment failure, underscoring the need for new treatment options that are active against resistant variants of the virus with a novel mechanism of action.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Lenacapavir is a breakthrough innovation with the potential to be a preferred and versatile foundational long-acting agent due to its therapeutic potency and a range of dosing frequencies and routes of administration. Lenacapavir is being developed as a foundation for Gilead’s future HIV therapies with the goal of offering several long-acting options that help address individual needs and preferences that may help optimize outcomes and reduce burden of care. Lenacapavir is being studied in multiple ongoing early and late-stage development programs and has the potential to offer a diverse set of person-centric options for treatment and prevention that could uniquely fit into the lives of people living with HIV and people who would benefit from pre-exposure prophylaxis (PrEP). The use of lenacapavir for HIV prevention is investigational and the safety and efficacy of lenacapavir for this use have not been established.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"></p><blockquote>“This news is an important milestone in the work to help end the HIV epidemic as Sunlenca is now the only FDA-approved twice-yearly treatment for people with multi-drug resistant HIV,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. “Gilead scientists have developed a unique and potent antiretroviral medicine in Sunlenca with the potential for flexible dosing options. Our goal is to deliver multiple long-acting options for treatment and prevention that are tailored to the needs of people living with HIV and people who could benefit from PrEP medicines.”</blockquote><p></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The FDA approval for Sunlenca is supported by data from the Phase 2/3 CAPELLA trial, which evaluated lenacapavir in combination with an optimized background regimen in people with multi-drug resistant HIV-1 who are heavily treatment experienced. CAPELLA participants had undergone previous treatment with a median of nine antiretroviral medications. In this patient population with significant unmet medical need, 83% (n=30/36) of participants randomly allocated to receive lenacapavir in addition to an optimized background regimen achieved an undetectable viral load (<50 copies/mL) at Week 52. Additionally, these participants achieved a mean increase in CD4 count of 82 cells/µL. These data were presented at the 29th Conference on Retroviruses and Opportunistic Infections (virtual CROI 2022).</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Sunlenca was reviewed and approved as a medication with FDA Breakthrough Therapy Designation, which is intended to expedite the development and review of new drugs which may demonstrate substantial improvement over available therapy. In May 2019, the FDA granted Breakthrough Therapy Designation for the development of lenacapavir for the treatment of HIV-1 infection in heavily treatment-experienced patients with multi-drug resistance in combination with other antiretroviral drugs.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Lenacapavir, alone or in combination, is not approved by any regulatory authority outside of the United States, United Kingdom, Canada or the European Union for any use. The European Marketing Authorization applies to all 27 member states of the European Union, as well as Norway, Iceland and Liechtenstein.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">The use of lenacapavir for HIV prevention is investigational and the safety and efficacy of lenacapavir for this use have not been established. Gilead is studying the safety and efficacy of lenacapavir for HIV prevention in multiple ongoing clinical studies.</p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;">Ref : https://en.wikipedia.org/wiki/Lenacapavir</p><span><a name='more'></a></span><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-sunlenca-lenacapavir-twice-yearly-living-multi-resistant-hiv-5946.html" style="background-color: transparent;">FDA Approves Sunlenca (lenacapavir) Twice-Yearly Treatment for People Living With Multi-Drug Resistant HIV</a></p><p style="background-color: white; box-sizing: inherit; color: #202227; font-family: -apple-system, BlinkMacSystemFont, "Segoe UI", "Segoe UI Symbol", "Helvetica Neue", Roboto, Arial, sans-serif; font-size: 17px; margin: 12px 0px 20px; padding: 0px; text-align: justify;"><a href="https://www.drugs.com/newdrugs/fda-approves-sunlenca-lenacapavir-twice-yearly-living-multi-resistant-hiv-5946.html"></a></p><div style="text-align: justify;"><br /></div></div><div class="blogger-post-footer">Medicinal Chemistry, FDA Approval, New Drugs, Drug Discovery</div>https://www.med-chemist.comhttp://www.blogger.com/profile/00786134978315983333noreply@blogger.com0