FDA Approves Nereus (tradipitant) for the Prevention of Motion Sickness

Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration has approved Nereus (tradipitant), an oral neurokinin-1 (NK-1) receptor antagonist, for the prevention of vomiting induced by motion. This approval marks the first new pharmacologic treatment in motion sickness in over four decades, representing a significant advancement in the understanding and management of this debilitating physiologic response that affects a substantial portion of the population and has long been recognized as a factor affecting military operational readiness.

"This approval underscores the strong scientific evidence in the antiemetic effects of Nereus in motion sickness," said Mihael H. Polymeropoulos, M.D., President, CEO and Chairman of the Board of Vanda Pharmaceuticals. "For the first time in over 40 years, patients have access to a novel therapy grounded in modern neuropharmacology, offering effective prevention without the limitations of existing options. We are proud of this historic milestone and grateful to the Vanda researchers, patients, investigators, and regulators who contributed to this achievement."

The efficacy of Nereus is supported by robust data from three pivotal clinical trials—two Phase 3 real-world provocation studies conducted on boats (Motion Syros and Motion Serifos) and one additional supporting study—with participants who had documented histories of motion sickness. In Motion Syros (n=365), vomiting incidence was 18.3–19.5% with Nereus versus 44.3% with placebo (p<0.0001).1 In Motion Serifos (n=316), vomiting rates were 10.4–18.3% with Nereus versus 37.7% with placebo (p≤0.0014), representing risk reductions of over 50–70%.2 Across the pivotal program, Nereus consistently demonstrated significant reductions in vomiting and a favorable safety profile consistent with acute use.

Motion sickness has been recognized as a critical factor in military operations since World War II, most notably during the D-Day invasion of Normandy in 1944, where severe seasickness impaired the effectiveness of troops, including paratroopers of the 101st Airborne Division deployed in rough Channel crossings and airborne drops. The condition was elevated to a strategic imperative, prompting early research into antiemetic therapies to ensure operational readiness during large-scale troop deployments by sea, air, and land.

Today, motion sickness remains prevalent in civilian life, with approximately 25–30% of adults3 —roughly 65–78 million people in the U.S.—experiencing symptoms during common travel modes such as cars, planes, or boats. Globally, up to one-third of individuals are highly susceptible.4 While most cases are mild, an estimated 5–15% of the population experiences severe, recurrent symptoms that can significantly impact quality of life. This severe segment comprises two key groups: those whose illness is inadequately controlled by existing therapeutic options, leaving them with persistent debilitating symptoms despite treatment, and those whose illness is so severe that it leads to avoidance of engaging in motionprovoking activities altogether, resulting in altered travel plans, missed opportunities, or complete abstention from certain modes of transportation or experiences. Tens of millions seek pharmacologic treatment annually, primarily through over-the-counter options, though many patients opt for prescription therapies when escalating care. Motion sickness arises from a sensory conflict between visual, vestibular, and proprioceptive inputs, triggering the release of substance P and activation of NK-1 receptors in the central nervous system, leading to nausea and vomiting. Nereus's mechanism of action—potent and selective antagonism of NK-1 receptors—directly addresses this pathway.

The approval of Nereus for the prevention of vomiting induced by motion validates its pharmacological profile and paves the way for further exploration of NK-1 antagonism in related vomit-inducing conditions. Vanda is advancing tradipitant in clinical development for gastroparesis, a chronic disorder characterized by delayed gastric emptying and persistent nausea/vomiting, as well as for the prevention of nausea and vomiting induced by GLP-1 receptor agonists—a common side effect impacting adherence in the rapidly growing obesity and diabetes treatment landscape.

Vanda anticipates launching Nereus for the prevention of vomiting induced by motion in the coming months and remains committed to expanding its therapeutic potential across indications driven by substance P-mediated pathways.

https://en.wikipedia.org/wiki/Tradipitant

FDA Approves Nereus (tradipitant) for the Prevention of Motion Sickness

FDA Approves Zycubo (copper histidinate) for Children With Menkes Disease

Sentynl Therapeutics Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences Limited (“Zydus Group”), announced that the U.S. Food and Drug Administration (“FDA”) has approved Zycubo® (copper histidinate) for the treatment of Menkes disease in pediatric patients. This is the first and only treatment approved for Menkes disease, a rare and fatal genetic condition, in the United States. Zycubo® is not indicated for the treatment of Occipital Horn Syndrome.

“Approval is a pivotal step towards achieving our goal of making a meaningful impact on patients, caregivers, and the rare disease community,” said Dr. Sharvil P. Patel, Managing Director, Zydus Lifesciences Limited. “This milestone marks a transformative moment for the Zydus Group and for families affected by Menkes disease. For the first time, patients have access to an approved therapy, offering hope where no options existed. We are proud to advance patient care and deliver innovative solutions to those who need them most.”

Menkes disease is a rare X-linked recessive pediatric disease caused by mutations of the copper transporter ATP7A encoded by the ATP7A gene. Patients with Menkes disease are born with the inability to absorb dietary copper and subsequently have impaired copper transport across the blood-brain barrier, and, until now, there has been no approved treatment in the United States. Zycubo® is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in patients with Menkes disease.

“Menkes disease presents significant challenges for patients and their families. With no known cure, most untreated patients do not survive beyond three years of age,” said Matt Heck, CEO, Sentynl. “FDA’s approval serves as compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease.”

The approval is supported by positive topline clinical efficacy results for Zycubo, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (“ET”) with Zycubo, compared to an untreated contemporaneous external control cohort, with a nearly 80% reduction in the risk of death. Median overall survival (“OS”) was 177.1 months for Zycubo ET cohort compared to 17.6 months for the untreated contemporaneous external control cohort.

The most common adverse reactions (incidence ≥7%) were pneumonia, viral infection, respiratory failure, seizure, bacterial infection, hemorrhage, hypotension, vomiting, tachycardia, pyrexia, volume depletion, fracture, dyspnea, transaminases elevation, diarrhea, fungal infection, anemia, and local administration reaction.

“This milestone represents the culmination of decades of research into better understanding and ultimately finding an effective treatment for Menkes disease,” said Dr. Stephen Kaler, a clinical genetics and genomics specialist at the Columbia University Medical Center. “Increased awareness of Menkes disease and rapid testing upon suspicion are critical, as beginning copper histidinate therapy in affected neonates has been shown to reduce symptoms and prolong life.”

Acquired from Cyprium Therapeutics in 2023, Sentynl has advanced Zycubo through the final stages of development with the FDA based on positive results from pivotal studies, receiving Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and the FDA Orphan Drug Designations. Copper histidinate has also been granted Orphan Designation by the European Medicines Agency.

ttps://upload.wikimedia.org/wikipedia/commons/thumb/a/ae/Copper_histidinate_v2.svg/250px-Copper_histidinate_v2.svg.png

FDA Approves Zycubo (copper histidinate) for Children With Menkes Disease

FDA Approves Zycubo (copper histidinate) for Children With Menkes Disease

Sentynl Therapeutics Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences Limited (“Zydus Group”), announced that the U.S. Food and Drug Administration (“FDA”) has approved Zycubo® (copper histidinate) for the treatment of Menkes disease in pediatric patients. This is the first and only treatment approved for Menkes disease, a rare and fatal genetic condition, in the United States. Zycubo® is not indicated for the treatment of Occipital Horn Syndrome.

“Approval is a pivotal step towards achieving our goal of making a meaningful impact on patients, caregivers, and the rare disease community,” said Dr. Sharvil P. Patel, Managing Director, Zydus Lifesciences Limited. “This milestone marks a transformative moment for the Zydus Group and for families affected by Menkes disease. For the first time, patients have access to an approved therapy, offering hope where no options existed. We are proud to advance patient care and deliver innovative solutions to those who need them most.”

Menkes disease is a rare X-linked recessive pediatric disease caused by mutations of the copper transporter ATP7A encoded by the ATP7A gene. Patients with Menkes disease are born with the inability to absorb dietary copper and subsequently have impaired copper transport across the blood-brain barrier, and, until now, there has been no approved treatment in the United States. Zycubo® is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in patients with Menkes disease.

“Menkes disease presents significant challenges for patients and their families. With no known cure, most untreated patients do not survive beyond three years of age,” said Matt Heck, CEO, Sentynl. “FDA’s approval serves as compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease.”

The approval is supported by positive topline clinical efficacy results for Zycubo, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (“ET”) with Zycubo, compared to an untreated contemporaneous external control cohort, with a nearly 80% reduction in the risk of death. Median overall survival (“OS”) was 177.1 months for Zycubo ET cohort compared to 17.6 months for the untreated contemporaneous external control cohort.

The most common adverse reactions (incidence ≥7%) were pneumonia, viral infection, respiratory failure, seizure, bacterial infection, hemorrhage, hypotension, vomiting, tachycardia, pyrexia, volume depletion, fracture, dyspnea, transaminases elevation, diarrhea, fungal infection, anemia, and local administration reaction.

“This milestone represents the culmination of decades of research into better understanding and ultimately finding an effective treatment for Menkes disease,” said Dr. Stephen Kaler, a clinical genetics and genomics specialist at the Columbia University Medical Center. “Increased awareness of Menkes disease and rapid testing upon suspicion are critical, as beginning copper histidinate therapy in affected neonates has been shown to reduce symptoms and prolong life.”

Acquired from Cyprium Therapeutics in 2023, Sentynl has advanced Zycubo through the final stages of development with the FDA based on positive results from pivotal studies, receiving Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and the FDA Orphan Drug Designations. Copper histidinate has also been granted Orphan Designation by the European Medicines Agency.

FDA Approves Zycubo (copper histidinate) for Children With Menkes Disease

FDA Approves Yuvezzi (carbachol and brimonidine tartrate) Ophthalmic Solution to Treat Presbyopia

Tenpoint Therapeutics, Ltd., a global, commercial biotechnology company focused on developing groundbreaking treatments to improve vision in the aging eye, today announced that the U.S. Food and Drug Administration (FDA) approved Yuvezzi (carbachol and brimonidine tartrate ophthalmic solution) 2.75%/0.1%, previously known as Brimochol™ PF, the first and only dual-agent eye drop for the treatment of presbyopia in adults. Presbyopia, the gradual loss of near vision that typically begins around age 45, affects about two billion people globally and 128 million people in the U.S.3,4,5 Yuvezzi is expected to be broadly commercially available in the U.S. in Q2 2026.

Carbachol

brimonidine 

Yuvezzi (carbachol and brimonidine tartrate ophthalmic solution) 2.75%/0.1% isthe only dual-agent presbyopia-correcting eye drop intentionally designed to deliver durability, tolerability, and safety

Yuvezzi was studied in two pivotal Phase 3 trials enrolling more than 800 patients, including the world’s largest and longest safety study for presbyopia eye drops1,2

Yuvezzi achieves miosis from 30-minutes up to 10 hours with one drop daily1

The FDA approval of Yuvezzi is based on positive data from two Phase 3 studies. The Phase 3 BRIO I study demonstrated a superior benefit of the combination therapy over the individual actives – a requirement for FDA approval of a fixed-dose combination.1 In the second Phase 3 study, BRIO II, which was vehicle-controlled, Yuvezzi achieved all primary near vision improvement endpoints with statistically significant three-lines or greater improvement in binocular uncorrected near visual acuity (BUNVA) over 8 hours, without the loss of one line or more in binocular uncorrected distance visual acuity (BUDVA).2 In addition, Yuvezzi was well-tolerated with no treatment-related serious adverse events observed in the more than 72,000 treatment days monitored in BRIO II, the longest safety study (12 months) conducted in presbyopia to-date.2 The most common side effects of Yuvezzi are headache, impaired vision and temporary eye pain or eye irritation.6

Eye redness was not a commonly reported side effect in clinical trials of Yuvezzi. In BRIO I and BRIO II the reports of adverse events of ocular hyperemia (eye redness) were low.1,2 In BRIO II, the rate of reported adverse events of ocular hyperemia (eye redness) was lower in subjects receiving Yuvezzi (2.8%) than carbochol alone (10.7%).2

“The FDA approval of Yuvezzi represents a significant milestone for the millions of people in the U.S. living with presbyopia and its daily frustrations and challenges,” said Henric Bjarke, Chief Executive Officer of Tenpoint Therapeutics. “As the first FDA-approved dual-agent eye drop for presbyopia, Yuvezzi leverages the mechanisms of carbachol and brimonidine tartrate to deliver sharp near vision with favorable tolerability. People deserve treatments that not only work but also can fit conveniently into their daily lives, and Yuvezzi brings an innovative new option to the presbyopia category. This approval marks the first groundbreaking therapy for Tenpoint Therapeutics aimed at advancing our mission to bring innovation to the aging eye.”

“The impact of presbyopia is often underestimated, and current solutions like glasses, contacts or surgery have fallen short in meeting the real-world needs of people who struggle with close-up tasks,” said John Hovanesian, M.D., FACS, of Harvard Eye Associates in Laguna Hills, California. “Yuvezzi introduces a novel approach by combining carbachol and brimonidine tartrate in a single daily eye drop that sharpens near vision and maintains tolerability throughout the day. Yuvezzi was intentionally designed to deliver both efficacy and tolerability, which represents an important step forward in delivering a complete, noninvasive option for people with presbyopia.”

“Presbyopia remains a universal and progressive condition that requires individualized management strategies,” said Mile Brujic, OD, FAAO, of Premier Vision Group in Bowling Green, Ohio. “I’m excited that I will be able to offer my patients YuvezziTM, the first-of-its-kind eye drop well-suited for long-term use. For people seeking a reliable, lifestyle-friendly solution to manage their near vision challenges in aging eyes, Yuvezzi is a valuable and practical advancement.”

“We’ve been working diligently to bring Yuvezzi to the eye care community and to the millions of adults looking for the optimal solution to address their frustration with presbyopia,” said Carol Kearney, Chief Commercial Officer of Tenpoint Therapeutics. “It was important to us that we provide an option that fits seamlessly into active, social and professional lifestyles, and we intentionally designed Yuvezzi to offer the right balance of efficacy, tolerability and ease of use.”

https://en.wikipedia.org/wiki/Carbachol

https://go.drugbank.com/drugs/DB00484

FDA Approves Yuvezzi (carbachol and brimonidine tartrate) Ophthalmic Solution to Treat Presbyopia

FDA Approves Lynavoy (linerixibat) for Cholestatic Pruritus in Patients with Primary Biliary Cholangitis

GSK  announced   the US Food and Drug Administration (FDA)  approval of  Lynavoy (linerixibat) for the treatment of cholestatic pruritus in adult patients with PBC. Lynavoy, an ileal bile acid transporter (IBAT) inhibitor that reduces multiple drivers of chronic itch, is the first medicine approved in the US for this indication.5

Lynavoy, an ileal bile acid transporter (IBAT) inhibitor, is the first medicine approved in the US for the treatment of cholestatic pruritus in patients with PBC

Up to 89% of people living with PBC experience cholestatic pruritus, an internal itch with a debilitating impact on quality of life1-4

Approval based on the positive GLISTEN phase III trial with regulatory reviews underway in the EU, UK, Canada and China

GSK previously announced on 9 March a licence agreement under which Alfasigma S.p.A. will acquire worldwide exclusive rights to develop, manufacture and commercialise linerixibat. This transaction is ongoing and is subject to customary conditions, including applicable regulatory agency clearances such as under the Hart-Scott-Rodino Act in the US.

Cholestatic pruritus is an internal itch experienced by up to 89% of people living with PBC, a rare autoimmune disease that can lead to liver failure.1-4 It is a serious condition that can be debilitating, with patients experiencing sleep disturbance, fatigue, impaired quality of life and even sometimes requiring liver transplantation in the absence of liver failure

https://en.wikipedia.org/wiki/Linerixibat

FDA Approves Lynavoy (linerixibat) for Cholestatic Pruritus in Patients with Primary Biliary Cholangitis

FDA Approves Rhapsido (remibrutinib) for the Treatment of Chronic Spontaneous Urticaria

Novartis announced today that Rhapsido (remibrutinib) received US Food and Drug Administration (FDA) approval as an oral treatment for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment. Rhapsido is a pill taken twice daily and does not require injections or lab monitoring. It is the first FDA-approved Bruton's tyrosine kinase inhibitor (BTKi) for CSU. Rhapsido helps to inhibit the release of histamine and other proinflammatory mediators by targeting BTK, offering a unique approach to CSU treatment.

"CSU is a serious disease that can cause debilitating symptoms and unpredictable flares. It's difficult to diagnose and manage," said Mark Lebwohl, MD, Dean for Clinical Therapeutics at the Icahn School of Medicine at Mount Sinai and member of the steering committee for the remibrutinib REMIX Phase III clinical trial program. "Remibrutinib represents a new way of treating CSU. By blocking the activity of BTK, remibrutinib stops a key pathway of the immune response in CSU. This is an exciting new option that has the potential to help a broad range of patients get fast relief."

CSU is a mast cell-driven condition thought to be caused by immune dysregulation. In people with CSU, the immune system can become activated through allergic (IgE) or autoimmune (IgG) pathways.4 This causes certain immune cells—mast cells and basophils—to activate the BTK protein. While not fully understood, it is believed that once activated, BTK leads to the release of histamine and other proinflammatory mediators that may cause the red, swollen, and itchy hives commonly seen in CSU.5,6

CSU symptoms are unpredictable, recurring for six weeks or more without an identified cause.7 Diagnosis can take up to 24 months.8 Many CSU patients say their symptoms negatively impact their sleep, work, and mental health.9,10,11 Antihistamines are the first-line treatment, but over half of patients still have symptoms, even at higher doses.2 Injectable treatments exist for those who don't respond to antihistamines, yet fewer than 20% of eligible patients receive them.12

"The approval of remibrutinib is an important development in CSU care. It quickly reduces symptoms, offering patients control of the hives and itching that they experience on a daily basis," said Giselle Mosnaim, MD, MS, an Allergist and Immunologist from Endeavor Health, Clinical Associate Professor at the University of Chicago Pritzker School of Medicine and REMIX trial investigator. "This is significant because it expands beyond existing injectable treatments and gives patients an oral option that can easily be incorporated into their daily lives."

"Many CSU patients feel misunderstood and settle for treatments that don't fully meet their needs," said Lynda Mitchell, CEO of Allergy & Asthma Network. "We support new treatment options that empower patients to choose what works best for them. This convenient new oral therapy offers a promising new way to manage CSU and potentially improve daily life for those living with this challenging condition."

Clinical data supporting approval

The FDA approval of Rhapsido in CSU is based on results from the Phase III REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157) clinical trials in patients who remained symptomatic on second-generation H1 antihistamines. Rhapsido demonstrated superiority in change from baseline versus placebo in itch (ISS7), hives (HSS7), and weekly urticaria activity (UAS7) at Week 12.13 Significantly more patients treated with Rhapsido versus placebo achieved well-controlled disease (UAS7≤6) as early as Week 2 and at Week 12, and about one-third of patients achieved complete absence of itch and hives at Week 12.13 Rhapsido has a demonstrated safety profile that requires no lab monitoring.13 The most common adverse events (incidence ≥3%) were nasal congestion, sore throat, and runny nose (nasopharyngitis), bleeding, headache, nausea, and abdominal pain.13

Novartis has completed regulatory submissions for Rhapsido for the treatment of CSU across many countries, including in the European Union, Japan, and China, with priority review granted in China.

Transforming care in Immunology

"This approval of Rhapsido as the first and only BTK inhibitor in CSU is an important milestone in our journey to reshape care for overlooked immune-related conditions and offer more patients the potential to find fast relief," said Victor Bultó, President, US, Novartis. "Building on our legacy in advancing the treatment of allergic, dermatologic, and rheumatologic conditions, we are deeply committed to further investing in innovative, patient-focused therapies across immunology."

Discovered and developed by Novartis to target the BTK pathway as a driver of inflammation, remibrutinib is being investigated in ongoing clinical trials across a variety of immune-related conditions, including chronic inducible urticaria (CIndU), hidradenitis suppurativa (HS), and food allergy.

Rhapsido (remibrutinib) Indication

Rhapsido is a prescription medicine used to treat adults with chronic spontaneous urticaria (CSU) who continue to have symptoms that are not controlled with antihistamine treatment. Rhapsido should not be used to treat any other forms of hives (urticaria). It is not known if Rhapsido is safe and effective in children.

Rhapsido Important Safety Information

Before taking Rhapsido, patients should tell their health care provider about all of their medical conditions, including if they:

Have had a recent surgery or plan to have surgery. Health care providers may tell patients to stop taking Rhapsido for at least 3 to 7 days before and after any planned medical or surgical procedures

Have bleeding problems or are taking a blood thinner medicine

Have liver problems

Are pregnant or plan to become pregnant. It is not known if Rhapsido will harm an unborn baby. If patients become pregnant during treatment with Rhapsido, they should talk to their health care provider about registering in the pregnancy exposure registry for Rhapsido

Are breastfeeding or plan to breastfeed. It is not known if Rhapsido passes into breast milk

Have recently received or are scheduled to receive an immunization (vaccine). Patients should talk to their health care provider about receiving any immunizations before taking Rhapsido. They should not receive live or live-attenuated vaccines during treatment with Rhapsido

Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking Rhapsido with certain other medications may affect how Rhapsido or other medications work, and can cause side effects.

https://en.wikipedia.org/wiki/Remibrutinib

FDA Approves Rhapsido (remibrutinib) for the Treatment of Chronic Spontaneous Urticaria

FDA Approves Foundayo (orforglipron), the Only GLP-1 Pill for Weight Loss That Can be Taken Any Time of Day Without Food or Water Restrictions

Eli Lilly and Company (NYSE: LLY)  announced the FDA approval of   Foundayo™ (orforglipron) for adults with obesity, or overweight with weight-related medical problems. When used alongside a reduced-calorie diet and increased physical activity, Foundayo helps individuals lose excess body weight and keep the weight off. Foundayo will be available via LillyDirect®, with prescriptions accepted immediately and shipping beginning April 6, followed shortly after by broad availability through U.S. retail pharmacies and telehealth providers.

Adults taking Foundayo lost an average of 27 pounds on the highest dose in the ATTAIN-1 clinical trial1

Foundayo, Lilly's second FDA-approved obesity medicine, will be available via LillyDirect® with free home delivery, starting at $25 per month with commercial coverage and $149 for self-pay

"People living with obesity need treatment options that meet them where they are – and for many, a once-daily pill that can be taken with no food or water restrictions can offer them greater flexibility in how they approach their treatment," said Deborah Horn, DO, director of the Center for Obesity Medicine at McGovern Medical School at UTHealth Houston. "With Foundayo, we now have an oral option that delivered an average of 12.4% weight loss at the highest dose in clinical trials – addressing both the clinical realities of obesity and the practical challenges patients face every day."

Supported by the rigorous ATTAIN clinical trial program, Foundayo was proven to help people lose weight and keep it off. In the ATTAIN-1 trial, individuals taking the highest dose of Foundayo and who stayed on treatment lost an average of 27.3 pounds (12.4%) compared to 2.2 pounds (0.9%) with placebo.1 Participants taking Foundayo, regardless of trial completion, lost an average of 25 pounds (11.1%), compared to 5.3 pounds (2.1%) with placebo.2 In the ATTAIN program, Foundayo also led to reductions in many markers of cardiovascular risk, including waist circumference, non-HDL cholesterol, triglycerides and systolic blood pressure across all doses.

"Today, fewer than 1 in 10 people who could benefit from a GLP-1 are taking one, held back by access, stigma, perceived complexity or the belief that their condition isn't serious enough for treatment. We believe Foundayo can help level the playing field for those living with obesity or who are overweight and living with weight-related complications," said David A. Ricks, chair and CEO of Eli Lilly and Company. "As a convenient, once-daily oral pill that delivers meaningful weight loss, this is obesity care designed for the real world."

Lilly is committed to making Foundayo accessible and affordable. Eligible people with commercial insurance may pay as little as $25 per month with the Foundayo savings card.3 Individuals opting for self-pay can access Foundayo starting at $149 per month for the lowest dose. Additionally, eligible Medicare Part D individuals may be able to get Foundayo for $50 per month, beginning as soon as July 1, 2026.

Use of Foundayo with other GLP-1 receptor agonist medicines is not recommended. It is not known whether Foundayo is safe and effective for use in children. Foundayo may cause tumors in the thyroid, including thyroid cancer. Watch for possible symptoms, such as a lump or swelling in the neck, hoarseness, trouble swallowing or shortness of breath. If you have any of these symptoms, tell your health care provider. The most common side effects of Foundayo include nausea, constipation, diarrhea, vomiting, indigestion, stomach (abdominal) pain, headache, swollen belly, feeling tired, belching, heartburn, gas, and hair loss. These are not all the possible side effects of Foundayo.

"There is no single path that works for everyone living with overweight or obesity," said Joe Nadglowski, president and CEO of the Obesity Action Coalition. "New treatment options expand choice and help more people find care that fits their lives, their goals and where they are in their journey – whether they're just starting to explore treatment or looking for a different long-term approach."

Lilly has submitted orforglipron for weight management and/or type 2 diabetes in more than 40 countries and plans to launch in each country shortly after approval

https://en.wikipedia.org/wiki/Orforglipron

More: 

FDA Approves Foundayo (orforglipron), the Only GLP-1 Pill for Weight Loss That Can be Taken Any Time of Day Without Food or Water Restrictions

FDA Approves Jascayd (nerandomilast) to Treat Idiopathic Pulmonary Fibrosis

Boehringer Ingelheim’s Jascayd (nerandomilast) tablets has been approved by the U.S. Food and Drug Administration (FDA) as an oral treatment option for idiopathic pulmonary fibrosis (IPF) in adult patients.2 Jascayd is the first and only preferential inhibitor of phosphodiesterase 4B (PDE4B) to be approved in this indication. This represents a novel mechanism of action that exerts both antifibrotic and immunomodulatory effects, thereby slowing the decline in lung function in IPF patients.2,3

“This milestone represents a new era in the treatment of IPF, a rare and debilitating chronic condition that worsens lung function. Nerandomilast has proven to slow lung function decline in IPF,” said Toby Maher, M.D., Ph.D., Professor of Clinical Medicine, Keck School of Medicine, USC Los Angeles. “Nerandomilast is a welcome new treatment option with a well-tolerated safety profile for physicians to consider for appropriate patients.”

The FDA approval is based on data from two clinical trials: FIBRONEER™-IPF (NCT05321069) and Trial 2 (NCT04419506). The primary endpoint in FIBRONEER™-IPF was the absolute change from baseline in Forced Vital Capacity (FVC), a measure of lung function,4 in mL at week 52.2,3 Nerandomilast demonstrated a significantly smaller FVC decline compared to placebo.2,3 Specifically, the adjusted mean decline in patients receiving 18 mg or 9 mg nerandomilast was -106 mL and -122 mL, respectively, versus -170 mL in the placebo group.2 Additionally, a treatment effect was shown as early as week two with nerandomilast 18 mg compared to placebo, with changes from baseline in FVC continuing to diverge over time to week 52.2,3

“The FDA approval of nerandomilast is a pivotal moment for people living with IPF as this marks the first time in over 10 years that the treatment landscape is evolving,” said Shashank Deshpande, Chairman of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “This new step forward, driven by the compelling results of the FIBRONEER™-IPF trial, underscores our unwavering commitment to change the way we treat IPF by delivering innovative therapies like nerandomilast.”

The most common (≥5%) adverse reactions reported in patients treated with nerandomilast and more frequently than the placebo group were as follows for nerandomilast 18 mg, 9 mg and placebo, respectively: diarrhea (42%, 31%, 17%), COVID-19 (13%, 16%, 12%), upper respiratory tract infection (13%, 11%, 10%), depression (12%, 11%, 10%), weight decreased (11%, 10%, 8%), decreased appetite (9%, 9%, 5%), nausea (8%, 9%, 7%), fatigue (7%, 8%, 6%), headache (7%, 6%, 5%), vomiting (6%, 5%, 5%), back pain (6%, 5%, 4%) and dizziness (5%, 6%, 5%).2

Discontinuation due to adverse reactions occurred more frequently in patients treated with nerandomilast (with or without background antifibrotic treatment) 18 mg (15%) and 9 mg (12%) compared to placebo (11%).2 The most frequent adverse reaction leading to discontinuation of nerandomilast 18 mg and 9 mg was diarrhea (6% and 2%, respectively).2

There is no ‘Warnings and Precautions’ section in the FDA approved product label.2

“The FDA approval of nerandomilast is exciting news for people living with idiopathic pulmonary fibrosis and their caregivers,” said Scott Staszak, President and CEO of the Pulmonary Fibrosis Foundation. “There has been a long-standing need for new treatment options for IPF within our community and nerandomilast provides an important addition to the care landscape.”

https://en.wikipedia.org/wiki/Nerandomilast

FDA Approves Jascayd (nerandomilast) to Treat Idiopathic Pulmonary Fibrosis

FDA Approves Ferabright (ferumoxytol injection) Contrast Agent for Magnetic Resonance Imaging of the Brain

Azurity Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA)  approval of  Ferabright (ferumoxytol injection), the first and only iron-based contrast agent indicated for magnetic resonance imaging (MRI) of the brain in adults with known or suspected malignant neoplasms in the brain to visualize lesions with a disrupted blood-brain barrier.1

“Ferabright is an advanced superparamagnetic iron oxide nanoparticle contrast agent engineered for high relaxivity,” said Ronald Scarboro, CEO of Azurity Pharmaceuticals. “It significantly enhances image contrast and precision in brain tumor delineation compared to non-contrast MRI, offering clinicians a new tool for diagnostic imaging and improved patient care.”

In a clinical trial, Ferabright-enhanced MRI significantly improved visualization of primary and secondary brain neoplasms compared with pre-contrast imaging.1 In addition to improved imaging, Ferabright offers an extended imaging window due to its long half-life, which may support flexible MRI protocols without the need for multiple contrast administrations.1 As an iron-based agent, Ferabright is processed through the body’s natural iron metabolism pathways,3,6,7 potentially reducing concerns related to long-term retention associated with other contrast agents.2,8 And unlike many other agents, Ferabright is suitable for patients with renal insufficiency,2,3,4 enabling access to contrast-enhanced MRI while reducing exposure to heavy metals found in other contrast agents.5

“Ferumoxytol expands our MRI toolkit and provides an option for patients who are either contraindicated for or decline gadolinium,” said Csanad Varallyay, MD, PhD, Neuroradiologist based in Portland, Oregon, with longstanding research experience in ferumoxytol-enhanced MRI.

Ferabright will be supplied in single-dose vials of 300 mg elemental iron per 10 mL (30 mg/mL) and 510 mg elemental iron per 17 mL (30 mg/mL) for intravenous infusion over at least 15 minutes.1 Ferabright is contraindicated in patients with known hypersensitivity to ferumoxytol, any of Ferabright’s components, or any other intravenous iron products. Reactions have included anaphylaxis.1

https://www.chemicalbook.com/ChemicalProductProperty_EN_CB92493638.htm

FDA Approves Ferabright (ferumoxytol injection) Contrast Agent for Magnetic Resonance Imaging of the Brain

FDA Approves Tepylute (thiotepa) Ready-to-Dilute Injectable Formulation to Treat Breast Cancer and Ovarian Cancer

Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s New Drug Application (NDA) for Tepylute, a ready-to-dilute formulation to treat breast and ovarian cancer in an easier to prepare, injectable product that enables dosing accuracy.

Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s New Drug Application (NDA) for Tepylute, a ready-to-dilute formulation to treat breast and ovarian cancer in an easier to prepare, injectable product that enables dosing accuracy.1

‘’This approval fulfills an unmet need by addressing the shortcomings and handling complexities of the current lyophilized powder formulation,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “We have taken a vital oncology drug and made it easier for oncology clinics and hospitals to use, while also reducing medical personnel exposure to a hazardous drug.”

Tepylute, formerly SH-105, is the third FDA-approved drug for Shorla, and a significant milestone for the company as it seeks approval for several cancer-fighting drugs for the U.S. market.

“The approval of Tepylute represents an important milestone for Shorla as our first in-house developed NDA,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology.

Tepylute is a liquid form of a well-established, standard of care oncology drug, thiotepa. The new formulation eliminates the need for complex and time-consuming reconstitution. It provides consistent dosing accuracy and allows for “just in time” preparation.2

“Among Tepylute’s many benefits, it removes the necessity to reconstitute which can introduce additional risks of drug preparation errors,” emphasized Rayna Herman, Chief Commercial Officer. “We look forward to providing an update on our launch plans for Tepylute in the near future.”

The American Cancer Society estimates that more than 300,000 women will be diagnosed with breast cancer in the U.S in 2024.3 About 19,680 women will be diagnosed with ovarian cancer in the United States.4

Shorla Oncology is currently marketing two products with a robust pipeline including SH-201, the first palatable oral liquid treatment for certain forms of leukemia and other cancers. In April, the company announced the FDA had accepted SH-201 for an NDA review with an expected action date of November 30, 2024.

‘’This approval fulfills an unmet need by addressing the shortcomings and handling complexities of the current lyophilized powder formulation,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “We have taken a vital oncology drug and made it easier for oncology clinics and hospitals to use, while also reducing medical personnel exposure to a hazardous drug.”

Tepylute, formerly SH-105, is the third FDA-approved drug for Shorla, and a significant milestone for the company as it seeks approval for several cancer-fighting drugs for the U.S. market.

“The approval of Tepylute represents an important milestone for Shorla as our first in-house developed NDA,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology.

Tepylute is a liquid form of a well-established, standard of care oncology drug, thiotepa. The new formulation eliminates the need for complex and time-consuming reconstitution. It provides consistent dosing accuracy and allows for “just in time” preparation.2

“Among Tepylute’s many benefits, it removes the necessity to reconstitute which can introduce additional risks of drug preparation errors,” emphasized Rayna Herman, Chief Commercial Officer. “We look forward to providing an update on our launch plans for Tepylute in the near future.”

The American Cancer Society estimates that more than 300,000 women will be diagnosed with breast cancer in the U.S in 2024.3 About 19,680 women will be diagnosed with ovarian cancer in the United States.4

Shorla Oncology is currently marketing two products with a robust pipeline including SH-201, the first palatable oral liquid treatment for certain forms of leukemia and other cancers. In April, the company announced the FDA had accepted SH-201 for an NDA review with an expected action date of November 30, 2024.

FDA Approves Tepylute (thiotepa) Ready-to-Dilute Injectable Formulation to Treat Breast Cancer and Ovarian Cancer

FDA Approves Ohtuvayre (ensifentrine) for the Maintenance Treatment of Chronic Obstructive Pulmonary Disease (COPD)

Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), announces the US Food and Drug Administration (“FDA”) approved Ohtuvayre (ensifentrine) for the maintenance treatment of chronic obstructive pulmonary disease (COPD) in adult patients. Ohtuvayre is the first inhaled product with a novel mechanism of action available for the maintenance treatment of COPD in more than 20 years.

Ohtuvayre is a first-in-class selective dual inhibitor of the enzymes phosphodiesterase 3 and phosphodiesterase 4 (“PDE3 and PDE4”) that combines bronchodilator and non-steroidal anti-inflammatory effects in one molecule. Ohtuvayre is delivered directly to the lungs through a standard jet nebulizer without the need for high inspiratory flow rates or complex hand-breath coordination.

“The approval of Ohtuvayre is a significant advance in COPD care, and we believe Ohtuvayre’s novel profile can change the treatment paradigm for COPD,” said David Zaccardelli, Pharm. D., President and Chief Executive Officer of Verona Pharma. “We plan to launch Ohtuvayre in the third quarter 2024, ensuring Ohtuvayre is available to help the millions of patients who still experience daily COPD symptoms.”

Michael Wells, MD, Associate Professor in the Division of Pulmonary, Allergy, and Critical Care Medicine at the University of Alabama Birmingham, commented: “In my experience, despite maintenance therapy, most patients report grappling with daily symptoms, including breathlessness and persistent coughing. COPD has a significant impact on both mortality and morbidity in the US, and until today, innovation in inhaled treatment modalities has been limited to combinations of existing treatment classes for over two decades. Ohtuvayre, as a first-in-class PDE3 and PDE4 inhibitor, offers a needed, unique approach and is an important advance in the treatment of COPD.”

The US approval of Ohtuvayre was based on extensive data including the Phase 3 ENHANCE trials, the results of which were published in the American Journal of Respiratory and Critical Care Medicine. In the ENHANCE trials, Ohtuvayre demonstrated clinical benefits both alone and when used with other maintenance therapies. Ohtuvayre was well-tolerated in a broad population of subjects with moderate to severe COPD.

The Company is fully staffed to launch and expects Ohtuvayre to be available in the third quarter 2024 through an exclusive network of accredited specialty pharmacies.

About Ohtuvayre (ensifentrine)

Ohtuvayre is the first inhaled therapy for the maintenance treatment of COPD that combines bronchodilator and non-steroidal anti-inflammatory activities in one molecule. Verona has evaluated nebulized Ohtuvayre in its Phase 3 clinical program ENHANCE (“Ensifentrine as a Novel inHAled Nebulized COPD thErapy”) for COPD maintenance treatment. Ohtuvayre met the primary endpoint in both ENHANCE-1 and ENHANCE-2, demonstrating statistically significant and clinically meaningful improvements in lung function. A fixed-dose combination of ensifentrine and glycopyrrolate, a LAMA, is currently under development for the maintenance treatment of COPD. Ensifentrine has potential applications for development in non-cystic fibrosis bronchiectasis, cystic fibrosis, asthma and other respiratory diseases.

REF: https://en.wikipedia.org/wiki/Ensifentrine

FDA Approves Ohtuvayre (ensifentrine) for the Maintenance Treatment of Chronic Obstructive Pulmonary Disease (COPD)

FDA Approves Sofdra (sofpironium) Topical Gel for the Treatment of Primary Axillary Hyperhidrosis

Clinical dermatology company, Botanix Pharmaceuticals Ltd. (ASX:BOT, Botanix or the Company), is pleased to announce the US Food and Drug Administration (FDA) approval of Sofdra™ (sofpironium) gel, 12.45%. Sofdra is a prescription medicine used to treat primary axillary hyperhidrosis (excessive underarm sweating) in adults and children 9 years and older.

Sofdra is the first and only new chemical entity approved by the FDA to treat primary axillary hyperhidrosis and presents a novel safe and effective solution for patients who have lacked treatment options for this socially challenging medical condition.

Botanix Chief Executive Officer, Dr Howie McKibbon, commented: "We are pleased to share this accomplishment with our dedicated Botanix team and dermatologist partners, patients who participated in the clinical studies and our shareholders who made this approval possible."

"This is a transformative event for Botanix as we transition from a development stage to a revenue generating dermatology company."

Hyperhidrosis is a condition characterised by abnormally increased sweating, beyond that required to regulate body temperature.1 The disproportionate sweat production that characterizes hyperhidrosis, results in a disabling medical condition with profound effects on the patient’s quality of life. Hyperhidrosis affects work productivity, daily routine activities, emotional well-being and personal relationships.2 Hyperhidrosis is the third largest dermatology condition (after acne and atopic dermatitis), with approximately 10 million patients in the US with primary axillary hyperhidrosis.3

David Pariser, MD, leading expert on hyperhidrosis, founding board member of the International Hyperhidrosis Society and past President of the American Academy of Dermatology commented: "The approval of Sofdra is terrific news for the hyperhidrosis community, which has been frustrated by the lack of effective and convenient treatment options."

"The availability of a new treatment alternative that is topical, well-tolerated, effective and easy to use is truly exciting and would be welcomed amongst patients and physicians."

The FDA approval of Sofdra was supported by results from the two pivotal Phase 3 ‘CARDIGAN’ studies which evaluated the efficacy and safety of Sofdra versus vehicle and enrolled 701 patients with primary axillary hyperhidrosis. In the studies, treatment with Sofdra successfully met all primary and secondary endpoints with clinically and statistically meaningful changes from baseline in Gravimetric Sweat Production (GSP) and the Hyperhidrosis Disease Severity Measure-Axillary, 7-item (HDSM-AX7) score.

An early patient experience program is planned to be launched by the Company in Q3 CY2024 to enable highly qualified patients to gain early access to Sofdra. These patients will be guided through the telemedicine and payer reimbursement process to be the first commercial users of the product. Broader launch of Sofdra is expected to follow in early Q4 CY2024 and Botanix expects to receive first revenues from sales in Q4 CY2024.

Botanix Executive Chairman, Mr Vince Ippolito, commented: "We are very excited to provide a new option for the 10 million patients with primary axillary hyperhidrosis in the United States."

"As the first and only new chemical entity, Sofdra represents a new therapeutic approach for dermatologists to treat patients with this disabling medical condition."

REF: https://en.wikipedia.org/wiki/Sofpironium_bromide

FDA Approves Sofdra (sofpironium) Topical Gel for the Treatment of Primary Axillary Hyperhidrosis

FDA Approves Vigafyde (vigabatrin) as the First and Only Ready-to-Use Vigabatrin Oral Solution

Pyros Pharmaceuticals, Inc. (Pyros or the Company), a leader in the development of enhanced specialty pharmaceuticals for rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has approved Vigafyde™, the only ready-to-use vigabatrin oral solution. Vigafyde™ (vigabatrin) oral solution, is indicated as a monotherapy for the treatment of pediatric patients 1 month to 2 years of age with infantile spasms (IS), where the potential benefits outweigh the potential risk of vision loss.  

Infantile spasms, a rare but severe form of epilepsy, poses significant challenges for patients and their families. Pyros is committed to providing dependable services and an affordable treatment option to help ensure that families facing an IS diagnosis, and whose children are prescribed vigabatrin can quickly obtain this essential therapy.

"The approval of Vigafyde™ marks the first new drug indicated for infantile spasms in fifteen years and underscores our unwavering commitment to supporting families facing this challenging condition," stated Michael Smith, co-founder and Chief Executive Officer of Pyros.

“This is a momentous day for patients and caregivers who have long awaited the first ready-to-use vigabatrin. I believe this approval will bring renewed energy and momentum to the infantile spasms community, enhancing our efforts to improve disease education, develop care pathways, and expand investment in infantile spasms research,” stated Edwin Urrutia, co-founder and Chief Operating Officer at Pyros.

Vigafyde™ is expected to be available in the second half of 2024. Additionally, Pyros Total Care™, the Company’s personalized comprehensive support program, is available to assist families throughout the treatment journey.

About Pyros Total Care™

As part of the Company’s commitment to prioritizing patient access to treatments for those who need them most, Pyros provides ongoing personalized support to caregivers through Pyros Total Care™ for those prescribed Vigafyde™. The support program offers personal assistance and financial resources to caregivers whose child is starting or continuing therapy.

Our dedicated support team includes a nurse educator, reimbursement support, and a clinical pharmacist. For more information, visit PyrosTotalCare.com or call 1-888-760-8330, Monday to Friday, 8 a.m. to 5 p.m. Central Time.

About Infantile Spasms

Infantile spasms (IS) is a rare, severe form of epilepsy that typically begins in children less than one year old.1 Infantile spasms can appear as subtle but repetitive movements that can often be overlooked or misdiagnosed. IS can lead to long-term permanent issues such as continued seizures, other forms of epilepsy, autism spectrum disorder, and developmental issues.2 The American Academy of Neurology conducted a systematic review of treatment for IS and concluded that successful treatment of IS can improve the long-term prognosis.3

About Vigabatrin

Vigabatrin is a medication used in the treatment of infantile spasms and is designed to inhibit the enzyme GABA transaminase, consequently increasing gamma-aminobutyric acid (GABA) levels in the brain.4,5 This release is thought to enhance seizure control for patients by modulating neuronal excitability.6 Vigabatrin's mechanism of action underscores its importance in treating seizure disorders.7 Its impact may extend beyond seizure management, with emerging research indicating potential benefits in mitigating neurodevelopmental complications associated with certain epilepsy syndromes

FDA Approves Vigafyde (vigabatrin) as the First and Only Ready-to-Use Vigabatrin Oral Solution

FDA Approves Lumisight (pegulicianine) Optical Imaging Agent to Illuminate Residual Breast Cancer Post-Lumpectomy

Lumicell, Inc., a privately held company focused on developing innovative fluorescence-guided imaging technologies for cancerous tissue detection during surgery, today announced the U.S. Food & Drug Administration (FDA) approved the company’s New Drug Application (NDA) for its Lumisight™ (pegulicianine) optical imaging agent and its Premarket Approval (PMA) application for Lumicell™ Direct Visualization System (DVS), together referred to as LumiSystem™.

With 84% diagnostic accuracy, LumiSystem enables surgeons to scan the breast cavity post-lumpectomy, in real-time, to detect and resect residual cancer that may have otherwise been missed, potentially sparing some patients from second surgeries.1-2 The LumiSystem combination is indicated for fluorescence imaging in adults with breast cancer as an adjunct for the intraoperative detection of cancerous tissue within the resection cavity following removal of the primary specimen during lumpectomy surgery.

“We are immensely proud of the dual approval of Lumisight and Lumicell DVS – we believe this is the first drug-device combination product approved in over a decade to have followed both of the FDA's most stringent NDA and PMA review processes,”3 said Howard Hechler, President and Chief Operating Officer, Lumicell. “With the FDA’s approval, LumiSystem is now the first and only imaging combination product capable of detecting cancerous tissue where it matters most, inside the breast cavity.”

“Breast cancer is all too common, and sadly, 1 in 8 women will develop it during their lifetime,”4 said Kelly Hunt, MD, Chair of the Department of Breast Surgical Oncology at MD Anderson Cancer Center and President of the Society of Surgical Oncology. “Our most common surgical procedure to treat these women is lumpectomy. Unfortunately, the intraoperative tools we have are limited and do not identify the extent of tumor accurately enough, making it challenging to achieve a complete tumor resection, leading to as many as 36% of patients needing a second surgery.”5

“Up to 65% of the time, we do not find residual cancer in the second surgery and are left wondering if we performed an unnecessary surgery due to a false positive margin assessment or if the cancer was missed again in the second surgery,”6 said Irene Wapnir, MD, Breast Surgical Oncologist and Professor of Surgery, Stanford University School of Medicine.

“During lumpectomy surgery, surgeons still struggle to identify and remove all of the tumor during the first operation,”7 said Barbara Smith, MD, PhD, Director of the Breast Program at Massachusetts General Hospital and Professor of Surgery at Harvard Medical School. “With LumiSystem, we will now have a technology that is clinically proven to achieve a more complete cancer resection during lumpectomy that could help some patients avoid a second surgery.”2

The safety of the system was established using data from more than 700 breast cancer patients across five clinical studies at top academic and regional community cancer centers across the U.S. The most common side effects with Lumisight are hypersensitivity and an abnormal color in urine. Lumisight may cause serious hypersensitivity reactions, including anaphylaxis. Results of the Investigation of Novel Surgical Imaging for Tumor Excision (INSITE) pivotal trial, used to support the efficacy of the system, were published in NEJM Evidence.

“We want to thank our clinical investigators and the hundreds of women who participated in our breast program for Lumisight and Lumicell DVS,” said Jorge Ferrer, Chief Scientific Officer, Lumicell. “Due to your important contributions, Lumisight and Lumicell DVS are now approved and will be available in the United States shortly.”

FDA Approves Lumisight (pegulicianine) Optical Imaging Agent to Illuminate Residual Breast Cancer Post-Lumpectomy