FDA Approves Seglentis (celecoxib and tramadol hydrochloride) for the Management of Acute Pain

In continuation of my update on celecoxib and tramadol

The U.S. Food and Drug Administration (FDA) has approved Seglentis (celecoxib and tramadol hydrochloride), a proprietary product developed by Esteve Pharmaceuticals' R&D team. It is an innovative first-in-class product comprised of a co-crystal form of celecoxib (an anti-inflammatory) and tramadol (an analgesic) for the treatment of acute pain in adults. This is Esteve's first proprietary research product to enter the United States market.

In words of Dr. Carlos Plata-Salamán, Chief Scientific Officer and Chief Medical Officer of Esteve "This innovation is the result of applying a crystallization technology to improve the physicochemical properties and pharmacokinetic characteristics of its active pharmaceutical ingredients.1,2,4,7,9 The FDA approval means that clinicians and adult patients in the U.S. now have a new treatment option for acute pain management."

Seglentis is the trade name for tablets that contain a co-crystal7 composed of celecoxib and tramadol hydrochloride. It is a new analgesic designed for acute pain management in a multimodal treatment approach3,5,6 targeting four complementary pain relief mechanisms.5,6 It offers a new treatment option for acute pain management aligned with the multimodal analgesia now considered standard of care. 

The novel co-crystal structure produces a unique pharmacokinetic profile of its active pharmaceutical ingredients compared to their individual or combined administration.1,2,4,9 The New Drug Application (NDA) was approved by the U.S. FDA on October 15, 2021.

Staffan Schüberg, Chief Executive Officer of Esteve, said: "We are proud of this milestone as we understand it as a recognition of our daily efforts to meet patient’s needs and to address the challenges the pain community is facing nowadays".

FDA Approves Zimhi (naloxone hydrochloride) Injection for the Treatment of Opioid Overdose

In continuation of my update on naloxone hydrochloride, Adamis Pharmaceuticals Corporation (Nasdaq: ADMP) announced   the U.S. Food and Drug Administration (FDA)   approval of  Adamis’ Zimhi™ (naloxone HCL Injection, USP) 5 mg/0.5 mL product. Zimhi is a high-dose naloxone injection product FDA-approved for use in the treatment of opioid overdose.

Naloxone is an opioid antagonist and is generally considered the drug of choice for immediate administration for opioid overdose. It works by blocking or reversing the effects of the opioid, including extreme drowsiness, slowed breathing, or loss of consciousness. Common opioids include morphine, heroin, tramadol, oxycodone, hydrocodone and fentanyl.

According to statistics published by the Centers for Disease Control and Prevention (CDC), drug overdoses resulted in approximately 96,779 deaths in the United States during the 12-month period ending March 2021, which was a 29% increase over the prior 12-month period. Drug overdoses are now the leading cause of death for Americans under age 50, with more powerful synthetic opioids, like fentanyl and its analogues, responsible for the largest number of those deaths.

Dr. Jeffrey Galinkin, an anesthesiologist, and former member of the FDA Advisory Committee for Anesthetics, Analgesics and Addiction Products, commented, “I am pleased to see this much needed high dose naloxone product will become part of the treatment tool kit as a countermeasure to the continued surge in fentanyl related deaths. The higher intramuscular doses of naloxone in Zimhi should result in more rapid and higher levels of naloxone in the systemic circulation, which in turn, should result in more successful resuscitations.”

Dr. Dennis J. Carlo, President and CEO of Adamis, stated, “We are very excited by this approval and are working with our commercial partner, US WorldMeds, to make this much-needed, lifesaving product readily available to the market. Zimhi provides the highest systemic levels of naloxone compared to any of the nasal or intramuscular products currently available.”

P. Breckinridge Jones, Sr., CEO of US WorldMeds, added, “We are pleased with the approval and now look forward to commercially marketing Zimhi in the United States. US WorldMeds has a proven track-record of successfully commercializing pharmaceutical products and have a First-in-Class and only FDA-approved product, LUCEMYRA® (lofexidine), for the treatment of withdrawal symptoms associated with abrupt opioid discontinuation. We are confident we can leverage our existing commercial infrastructure and presence in the opioid dependence market to speed the uptake of Zimhi and combat the growing opioid crisis. We are preparing for the full commercial launch of ZIMHI which is planned for the first quarter of 2022.”\

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp

Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions,   announced  the U.S. Food and Drug Administration (FDA)   approval of Klisyri (tirbanibulin) for the topical treatment of actinic keratosis (AK) on the face or scalp. Klisyri is the first FDA approved branded proprietary product for Athenex and will be launched in partnership with Almirall in the U.S. during the first quarter of 2021. Klisyri will be manufactured by Athenex, highlighting the vertically integrated capabilities of the company ranging from a preclinical lead to a developed product for market launch.

“The FDA approval of Klisyri is a significant milestone for Athenex. Klisyri is a home-grown product discovered and characterized by Athenex scientists and developed from pre-IND to NDA by the Athenex team. We are extremely proud of our team’s excellent execution,” said Dr. Johnson Lau, Chairman and Chief Executive Officer of Athenex. “Approval demonstrates our ability to execute upon the entirety of the drug development and registration process. We are excited to partner with Almirall to bring this first-in-class microtubule inhibitor to patients with actinic keratosis in the US.”

Dr. Rudolf Kwan, Chief Medical Officer of Athenex added, “The FDA approval of tirbanibulin ointment represents a first-in-class microtubule inhibitor for the treatment of actinic keratosis. We believe this small molecule platform has the potential beyond actinic keratosis and are leveraging the platform to develop therapies for other oncology indications.”

Mr. Peter Guenter, CEO of Almirall, stated, “We are delighted to partner with Athenex to market Klisyri in the U.S. and in Europe. This approval from the FDA represents a new option for Dermatologists and marks an important further step for Actinic Keratosis patients. What makes this new therapy particularly exciting is the 5-day course of treatment and its good tolerability. We look forward to the launch of Klisyri in the US in the first quarter of 2021.”

The FDA approved Klisyri based on the data from two pivotal, randomized, double-blind, vehicle-controlled Phase III studies (KX01-AK-003 and KX01-AK-004) that evaluated the efficacy and safety of Klisyri (tirbanibulin) ointment 1% in 702 adults with actinic keratosis of the face or scalp. Tirbanibulin demonstrated complete clearance of actinic keratosis lesions at day 57 in treated face or scalp areas in a significantly higher number of patients compared to vehicle. The most common adverse events were application site pruritus and pain reported by 9% and 10% of treated patients, respectively.

Actinic keratosis is a pre-cancerous skin lesion and is the second most common diagnosis made by dermatologists in the United States. If left untreated, 10-15% of AK lesions will develop into skin cancers.

Athenex has partnered with Almirall (Almirall, S.A., BME: ALM) to market Klisyri for the treatment of actinic keratosis on the face or scalp in the US and EU (including Russia) markets. In addition to the partnership with Almirall, Athenex has partnered with PharmaEssentia (6446.TWO) for actinic keratosis in Taiwan and has partnered with Xiangxue Pharmaceuticals (SHE:300147) for actinic keratosis in China, Hong Kong, and Macau.

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

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp

FDA Approves Orgovyx (relugolix) as the First Oral Gonadotropin-Releasing Hormone (GnRH) Receptor Antagonist for Advanced Prostate Cancer

Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women and for men, today announced that the U.S. Food and Drug Administration (FDA) has approved Orgovyx (relugolix) for the treatment of adult patients with advanced prostate cancer. Orgovyx, which was granted Priority Review by the FDA, is the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist for men with advanced prostate cancer. The approval is based on efficacy and safety data from the Phase 3 HERO study of Orgovyx in men with advanced prostate cancer. Orgovyx is expected to be available in January 2021.

“I am enormously pleased by the approval of Orgovyx and believe it has the potential to usher in a new standard of care for men with prostate cancer requiring androgen deprivation therapy,” said Neal Shore, M.D., medical director of the Carolina Urologic Research Center and HERO program steering committee member. “For the first time, we now have a once-daily oral treatment that effectively and rapidly suppresses testosterone, with a safety analysis showing a lower incidence of major adverse cardiovascular events compared to leuprolide injections, the current standard of care, as evaluated in the Phase 3 HERO study. The COVID-19 pandemic has heightened the importance of oral treatments as men with prostate cancer continue to experience difficulties and risks traveling to receive injections.”

“Prostate cancer is a very personal journey, but a universal truth is that those of us living with this disease want better treatments and options. That is why the approval of Orgovyx is such an exciting milestone that brings a long-awaited oral treatment option to men with advanced prostate cancer,” said Thomas Farrington, president and founder of the Prostate Health Education Network. “It is so important for men to speak with their doctor and explore what treatment is right for them as they focus on their overall health.”

“With the approval of Orgovyx, men with advanced prostate cancer now have a new oral treatment option that has demonstrated robust efficacy and safety, all with one pill taken once-a-day,” said Lynn Seely, M.D., chief executive officer of Myovant Sciences, Inc. “We have successfully built our commercial capabilities to bring this newly approved treatment to the urologists and oncologists who care for men with advanced prostate cancer, with the goal of establishing Orgovyx as the new standard of care. We are incredibly grateful to the men and investigators who participated in the HERO study and to the FDA for expediting the review and approval of Orgovyx through its Priority Review pathway.”

In the Phase 3 HERO study, Orgovyx met the primary endpoint and achieved sustained testosterone suppression to castrate levels (< 50 ng/dL) through 48 weeks in 96.7% (95% confidence interval [CI]: 94.9-97.9) of men, compared with 88.8% (95% CI: 84.6-91.8) of men receiving leuprolide acetate injections, the current standard of care. Orgovyx also achieved several key secondary endpoints compared to leuprolide acetate, including suppression of testosterone to castrate levels at Day 4 and Day 15 (56% versus 0% and 99% versus 12%, respectively) and profound suppression of testosterone (< 20 ng/dL) at Day 15 (78% versus 1%). Orgovyx lowered prostate-specific antigen (PSA), on average, by 65% at Day 15 and by 83% at Day 29. In a substudy, 55% of men treated with Orgovyx achieved normal testosterone levels (> 280 ng/dL) or returned to baseline within 90 days of treatment discontinuation. The most frequent adverse events reported in at least 10% of men in the Orgovyx group were hot flush, musculoskeletal pain, fatigue, constipation, and mild to moderate diarrhea. The HERO data were previously presented in an oral presentation at the 2020 American Society of Clinical Oncology (ASCO) Virtual Scientific Program, with simultaneous publication in the New England Journal of Medicine.

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

FDA Approves Orgovyx (relugolix) as the First Oral Gonadotropin-Releasing Hormone (GnRH) Receptor Antagonist for Advanced Prostate Cancer

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, announced  the U.S. Food and Drug Administration (FDA)  approval of  Zokinvy (lonafarnib) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies (PL).  

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients.  Disease manifestations include growth failure, loss of body fat and hair, aged-looking skin, stiffness of joints, hip dislocation, generalized atherosclerosis, cardiovascular disease and stroke.  Untreated children with Progeria die of heart disease at an average age of 14.5 years.  There are 20 children and young adults with Progeria and PL identified and followed in the U.S.  

Zokinvy is a disease-modifying agent that has demonstrated a statistically significant survival benefit in children and young adults with Progeria.  In patients with Progeria, Zokinvy reduced the incidence of mortality by 60% (p=0.0064) and increased average survival time by 2.5 years.  The most commonly reported adverse reactions were gastrointestinal (vomiting, diarrhea, nausea), and most were mild or moderate (Grade 1 or 2) in severity.  Many Progeria patients have received continuous Zokinvy therapy for more than 10 years.

The increase in survival observed with Zokinvy was derived from two open-label clinical trials (N=62) conducted at Boston Children's Hospital.  The survival analysis compared Zokinvy-treated versus Zokinvy-naïve subjects with Progeria born in or after 1991, by age, gender, and geographic location. Zokinvy-naïve patients originated from a separate natural history study (n=81) conducted by The Progeria Research Foundation.

With this approval, the FDA issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Eiger.  The Rare Pediatric Disease Priority Review Voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.  Eiger plans to sell the PRV and under the terms of the Collaboration and Supply Agreement with the Progeria Research Foundation (PRF) will share the proceeds equally with PRF.

"The FDA approval of Zokinvy is the result of a pioneering partnership between Eiger BioPharmaceuticals and PRF to bring the first approved therapy to children, young adults and families living with this devastating disease," said David Cory, President and CEO of Eiger.  "We are very proud that the first drug approval at Eiger confers a survival benefit to patients with one of the most ultra-rare, and ultimately fatal, pediatric diseases.  We are extremely grateful to all the children, young adults and their families who have made this possible through participation in the Zokinvy clinical trials."

"The approval of this breakthrough therapy is a critical milestone for the Progeria community and also for Eiger," said Thomas Dietz, PhD, Chairman of the Board at Eiger.  "The Eiger Board congratulates and commends the management team for their incredible dedication leading the company through its first NDA filing and approval, a major accomplishment for Eiger."

PRF Medical Director, Leslie Gordon, MD, PhD, added, "Shortly after our son, Sam, was diagnosed with Progeria, my family and I founded The Progeria Research Foundation to find the cause, treatments, and cure for all children with this fatal disease.  This first approved medication is a truly incredible milestone for the Progeria community as we forge ahead toward finding the cure.  We are thrilled to have Eiger as a partner in bringing Zokinvy to the approval finish line, and for their commitment to ensuring patient access to Zokinvy moving forward."

In support of the patient and healthcare provider community, Eiger is launching our dedicated service center, Eiger OneCare™.  This specialized team will offer personalized support, financial assistance, and access to Zokinvy, all designed for Progeria and processing-deficient Progeroid Laminopathy patients.  Eiger OneCare™ will be available Monday through Friday from 9 AM to 5 PM Eastern Time at 1-833-MYEIGER (1-833-693-4437).

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

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

FDA Approves Eysuvis (loteprednol etabonate) Ophthalmic Suspension for the Short-Term Treatment of the Signs and Symptoms of Dry Eye Disease

Kala Pharmaceuticals, Inc. (NASDAQ:KALA), a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies for diseases of the eye,  announced   the U.S. Food and Drug Administration (FDA) approval of Eysuvis (loteprednol etabonate ophthalmic suspension) 0.25% for the short-term (up to two weeks) treatment of the signs and symptoms of dry eye disease.

“The FDA approval of Eysuvis as the first prescription therapy specifically developed to address the short-term treatment needs of people living with dry eye disease is a major accomplishment for Kala and an important moment for patients, who have been waiting for an FDA-approved, safe, effective and fast-acting therapy,” said Mark Iwicki, Chairman, President and Chief Executive Officer of Kala Pharmaceuticals. “As we prepare to launch Eysuvis, we will leverage our strong foundation of highly experienced ophthalmology marketing, sales and market access professionals with the goal of establishing Eysuvis as the preferred, first-line prescription therapy for dry eye disease. We’d like to thank the many patients and investigators that were involved in the clinical trials that led to this important milestone.”

Dry eye disease is a chronic, episodic, multifactorial disease affecting the tears and ocular surface, and can involve tear film instability, inflammation, discomfort, visual disturbance and ocular surface damage. Approximately 80 percent of people living with dry eye disease suffer from episodic flares. These flares can be caused by a wide variety of triggers and often cannot be adequately managed with current therapies.

Eysuvis utilizes Kala’s AMPPLIFY® mucus-penetrating particle (MPP) Drug Delivery Technology to enhance penetration of loteprednol etabonate (LE) into target tissue on the ocular surface. LE targets the immune responses that drive acute dry eye disease flares. Prior to Eysuvis, there were no FDA-approved ocular corticosteroids for the treatment of dry eye disease. Kala Pharmaceuticals plans to launch Eysuvis in the U.S. by year-end.

“The approval of Eysuvis ushers in a new era in the treatment of dry eye disease and offers promise to the millions of dry eye patients who experience acute exacerbations, or flares, of their disease each year,” said Edward Holland, M.D., Director of Cornea Services at Cincinnati Eye Institute and Professor of Ophthalmology at the University of Cincinnati. “For the first time we will be able to offer dry eye patients a therapeutic option that provides rapid relief for both the signs and symptoms of the disease and that is safe and well tolerated.”

“Dry eye disease can significantly decrease quality-of-life among affected patients and drive decreased workplace productivity, contact lens intolerance and discontinuation, and poor cataract and refractory surgery outcomes,” said Kelly Nichols, O.D., M.P.H., Ph.D., F.A.A.O., Dean of the University of Alabama at Birmingham School of Optometry. “As the prevalence of dry eye disease increases, there is a tremendous need for new therapies to manage mild-to-moderate dry eye disease patients, many of whom currently go untreated. I am excited by the approval of EYSUVIS and confident that having access to an approved corticosteroid specifically for dry eye disease will meaningfully impact the management of patients across the U.S.”

The FDA granted approval to Eysuvis based on results from four clinical trials, including three Phase 3 trials and one Phase 2 trial, that demonstrated significant improvements in both the signs and symptoms of dry eye disease. Specifically, statistical significance was achieved after two weeks of dosing for the sign endpoint of conjunctival hyperemia in all three Phase 3 trials. Statistical significance was observed in two of the three Phase 3 trials for the symptom endpoints of ocular discomfort severity in both the overall intent-to-treat (ITT) population and in a predefined subgroup of ITT patients with more severe ocular discomfort at baseline. Eysuvis was well-tolerated across the four trials, with adverse events and intraocular pressure increases comparable to that observed with vehicle.

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

FDA Approves Eysuvis (loteprednol etabonate) Ophthalmic Suspension for the Short-Term Treatment of the Signs and Symptoms of Dry Eye Disease

FDA Approves Alkindi Sprinkle (hydrocortisone oral granules) for Pediatric Adrenocortical Insufficiency

In continuation of my update on hydrocortisone 

Eton Pharmaceuticals, Inc (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercialising innovative treatments for rare pediatric diseases,  announced the U.S. Food and Drug Administration (FDA)   approval of  Alkindi Sprinkle (hydrocortisone) oral granules as replacement therapy for Adrenocortical Insufficiency (AI) in children under 17 years of age. Alkindi Sprinkle is the first and only FDA-approved granular hydrocortisone formulation for the treatment of adrenocortical insufficiency specifically designed for use in children.

“The FDA approval of Alkindi Sprinkle is a breakthrough for patients and caregivers treating pediatric adrenocortical insufficiency. We are excited to offer an FDA-approved product that enables low dosing and administration of hydrocortisone to pediatric patients,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “We look forward to making the product available to patients in the coming months.”

“For years, we heard from parents about their struggle to provide the right dose to their child,” said Dina M. Matos, Executive Director of the CARES Foundation. “We are thrilled the FDA has approved Alkindi Sprinkle for pediatric patients with Adrenocortical Insufficiency including patients with Congenital Adrenal Hyperplasia, a type of Adrenocortical Insufficiency.”

The FDA approval of Alkindi Sprinkle was supported by six clinical studies, including the first and only interventional Phase III study of oral hydrocortisone for Pediatric AI in neonates to children under eight years of age. Prior to the approval of Alkindi Sprinkle, oral hydrocortisone was only FDA-approved in tablet formulations of 5mg and stronger. Many pediatric patients require significantly lower doses and the flexibility of precision titration. Alkindi Sprinkle will be available in 0.5mg, 1mg, 2mg, and 5mg strengths, allowing clinicians greater flexibility to individualize dosing based on each patient’s needs in accordance with the instructions for dosage and administration.

Eton expects Alkindi Sprinkle to be commercially available in the fourth quarter of 2020.

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

FDA Approves Alkindi Sprinkle (hydrocortisone oral granules) for Pediatric Adrenocortical Insufficiency

FDA Approves Gavreto (pralsetinib) for the Treatment of Adults With Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY),  announced  the U.S. Food and Drug Administration (FDA) approval of Gavreto (pralsetinib) for the treatment of adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. This indication was approved under the FDA’s accelerated approval program based on data from the Phase I/II ARROW study. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations.

“The FDA approval of Gavreto for RET fusion-positive non-small cell lung cancer is an important step towards our goal of providing an effective treatment option for every person diagnosed with lung cancer, no matter how rare or hard-to-treat their type of disease,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “We remain committed to finding personalized treatment options for people with cancer based on specific genomic or molecular alterations, and we look forward to partnering with Blueprint Medicines to further explore the potential of Gavreto across multiple RET-altered tumor types.”

RET-activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and medullary thyroid cancer (MTC), and treatment options that selectively target these genetic alterations are limited. In NSCLC, RET fusions represent approximately 1-2% of patients. Biomarker testing for these fusions is the most effective way to identify people who are eligible for treatment with Gavreto.

The approval is based on the results from the Phase I/II ARROW study, in which Gavreto produced durable clinical responses in people with RET fusion-positive NSCLC with or without prior therapy, and regardless of RET fusion partner or central nervous system involvement. Gavreto demonstrated an overall response rate (ORR) of 57% (95% CI: 46%, 68%) and complete response (CR) rate of 5.7% in the 87 people with NSCLC previously treated with platinum-based chemotherapy, and the median duration of response (DoR) was not reached (95% CI: 15.2 months, not reached). In the 27 people with treatment-naïve NSCLC, the ORR was 70% (95% CI: 50%, 86%) with an 11% CR rate. The most common adverse reactions (≥25%) were fatigue, constipation, musculoskeletal pain and increased blood pressure (hypertension).

Gavreto is now the sixth FDA-approved medicine in Genentech’s portfolio of treatments for lung cancer. The FDA granted Breakthrough Therapy Designation to Gavreto for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy and for RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

The FDA has also granted Priority Review to Gavreto for the treatment of people with advanced or metastatic RET-mutant MTC and RET fusion-positive thyroid cancer, and is expected to make a decision on approval by February 28, 2021. This New Drug Application (NDA) was accepted for review under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

For those who qualify, Blueprint Medicines will offer patient assistance programs for people prescribed Gavreto by their doctor through YourBlueprint™ . Please visit www.yourblueprint.com or contact 1-888-BLUPRNT for more information.

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

FDA Approves Gavreto (pralsetinib) for the Treatment of Adults With Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer

FDA Approves Sesquient (fosphenytoin sodium) for the Treatment of Status Epilepticus in Adult and Pediatric Patients

Sedor Pharmaceuticals, LLC (Sedor) announced the U.S. Food and Drug Administration (FDA) spproval of  Sesquient (fosphenytoin sodium for injection) for the treatment of status epilepticus in adult and pediatric patients.

According to Neurocritical Care Society Guidelines, status epilepticus, which is classified as a single epileptic seizure lasting more than five minutes or two or more seizures within a five-minute period, must be treated quickly since irreversible brain damage or death may result if cessation of seizure is not achieved within 60 minutes of onset. Ready-to-dilute and room temperature stable, Sesquient is the only FDA-approved fosphenytoin that allows point-of-care storage, as well as fast and efficient administration in emergency rooms, intensive care units, first responder vehicles, and long-term care facilities, where serial seizures such as status epilepticus are most commonly treated.

“Status epilepticus is associated with irreversible neurologic damage and death, both of which largely depend on the seizure duration before initial treatment,” stated Barry Frankel, Chief Business Officer and co-founder of Sedor. “At some hospitals, it can take up to 30 minutes to get a status epilepticus drug from the pharmacy to the point of care in the ER to treat a patient. Sesquient – the first and only FDA-approved room temperature stable fosphenytoin – could help health care providers quickly treat status epilepticus patients and potentially reduce hospital costs associated with this condition.”

“This is an important milestone for Sedor Pharmaceuticals and opens a significant global market for the company. As our first NDA approved drug, Sesquient validates our business model of efficient development of critical care hospital injectable products,” added John Sedor, Chairman, CEO and co-founder of Sedor. Mr. Sedor added “While Sesquient is available in pre-filled liquid vials, this is a multi-dosage platform which we believe will be followed by a pre-filled IV bag. With this achievement completed, we are turning our focus to the development of our second product, Meloxicam for injection solubilized with betadex sulfobutyl ether sodium, for the potential treatment of acute post-surgical pain.”

Sedor is actively engaged in discussions to license the rights to Sesquient for North America, Europe, and other territories except for the Peoples Republic of China, where it has already been successfully licensed. Concurrently, Sedor is in discussions on securing capital to retain North American rights and commercialize the product.

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

FDA Approves Sesquient (fosphenytoin sodium) for the Treatment of Status Epilepticus in Adult and Pediatric Patients

FDA Approves Detectnet (copper Cu 64 dotatate injection) Positron Emission Tomography (PET) Agent

RadioMedix Inc. and its commercial partner Curium announced the approval of  Detectnet (copper Cu 64 dotatate injection)  by the U.S. Food and Drug Administration (FDA).  Detectnet is a positron emission tomography (PET) agent indicated for the localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult patients.  Curium expects to launch Detectnet immediately with doses available through various nuclear pharmacies or directly from Curium.   

RadioMedix Inc. and its commercial partner Curium announced today that Detectnet (copper Cu 64 dotatate injection) was approved by the U.S. Food and Drug Administration (FDA).  Detectnet is a positron emission tomography (PET) agent indicated for the localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult patients.  Curium expects to launch Detectnet immediately with doses available through various nuclear pharmacies or directly from Curium.   

“Detectnet brings an exciting advancement in the diagnosis of neuroendocrine tumors for healthcare providers, patients, and their caregivers,” said Ebrahim Delpassand, MD, CEO of RadioMedix.  “The Phase III results demonstrate the clinical sensitivity and specificity of Detectnet which will provide a great aid to clinicians in developing an accurate treatment approach for their NET patients.  Perhaps most exciting is that the 12.7-hour half-life allows Detectnet to be produced centrally and shipped to sites throughout the U.S.  This will help alleviate shortages or delays that have been experienced with other somatostatin analogue PET agents.” 

“Curium is excited to bring the first commercially available Cu 64 diagnostic agent to the U.S. market.  Our unique production capabilities and distribution network allow us to deliver to any nuclear pharmacy, hospital or imaging center its full dosing requirements first thing in the morning, to provide scheduling flexibility to the institution and its patients,” said Curium CEO, North America, Dan Brague.  “We look forward to joining with healthcare providers and our nuclear pharmacy partners to bring this highly efficacious agent to the market.”      

“Detectnet brings an exciting advancement in the diagnosis of neuroendocrine tumors for healthcare providers, patients, and their caregivers,” said Ebrahim Delpassand, MD, CEO of RadioMedix.  “The Phase III results demonstrate the clinical sensitivity and specificity of Detectnet which will provide a great aid to clinicians in developing an accurate treatment approach for their NET patients.  Perhaps most exciting is that the 12.7-hour half-life allows Detectnet to be produced centrally and shipped to sites throughout the U.S.  This will help alleviate shortages or delays that have been experienced with other somatostatin analogue PET agents.” 

“Curium is excited to bring the first commercially available Cu 64 diagnostic agent to the U.S. market.  Our unique production capabilities and distribution network allow us to deliver to any nuclear pharmacy, hospital or imaging center its full dosing requirements first thing in the morning, to provide scheduling flexibility to the institution and its patients,” said Curium CEO, North America, Dan Brague.  “We look forward to joining with healthcare providers and our nuclear pharmacy partners to bring this highly efficacious agent to the market.”      

https://en.wikipedia.org/wiki/Copper_(64Cu)_oxodotreotide

FDA Approves Detectnet (copper Cu 64 dotatate injection) Positron Emission Tomography (PET) Agent

FDA Approves Qdolo (tramadol hydrochloride) Oral Solution for the Management of Severe Pain

In continuation of my update on tramadol 

Bioscience, LLC, a specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved Qdolo™ (tramadol hydrochloride) Oral Solution 5mg/1mL C-IV, an opioid agonist indicated in adults for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate.

According to the Centers for Disease Control and Prevention, 50 million adults in the United States have chronic daily pain, with 19.6 million adults experiencing high-impact chronic pain that interferes with daily life or work activities. A May, 2019 U.S. Department of Health and Human Services report on pain management best practices emphasized the need for an "individualized, patient-centered" approach for the treatment of pain.1

"As an oral liquid, Qdolo gives physicians flexibility to titrate dosing precisely according to individual patients' needs," said Jeff Bryant, President and CEO of Athena Bioscience. "In addition, for patients with swallowing disfunction (dysphagia) or who simply have trouble swallowing pills, Qdolo provides an essential alternative to other forms of tramadol." Qdolo has a patent pending.

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

FDA Approves Qdolo (tramadol hydrochloride) Oral Solution for the Management of Severe Pain

FDA Approves Onureg (azacitidine tablets) as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia

Bristol Myers Squibb (NYSE: BMY) announced that the U.S. Food and Drug Administration (FDA) has approved Onureg (azacitidine 300 mg tablets, CC-486) for the continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are not able to complete intensive curative therapy.  AML is one of the most common acute leukemias in adults.

The approval is based on results from the pivotal Phase 3 QUAZAR® AML-001 study in which treatment with Onureg resulted in a statistically significant and clinically meaningful improvement in overall survival (OS), the study’s primary endpoint, of nearly 10 months compared to placebo. Median OS from time of randomization was greater than two years (24.7 months; 95% Confidence Interval [CI]: 18.7 to 30.5) among patients who received Onureg compared to 14.8 months (95% CI: 11.7 to 17.6) among patients receiving placebo (Hazard Ratio [HR]: 0.69, 95% CI: 0.55 to 0.86; p=0.0009). Onureg was continued until disease progression or unacceptable toxicity. Onureg has warnings and precautions for risks of substitution with other azacitidine products, myelosuppression, increased early mortality in patients with myelodysplastic syndromes (MDS) and embryo-fetal toxicity. Due to substantial differences in the pharmacokinetic parameters, Onureg should not be substituted for intravenous or subcutaneous azacitidine as it may result in a fatal adverse reaction. New or worsening Grade 3 or 4 neutropenia and thrombocytopenia occurred in 49% and 22% of patients who received Onureg, respectively. Febrile neutropenia occurred in 12% of patients. Complete blood counts should be monitored, dosing should be modified as recommended and standard supportive care should be provided if myelosuppression occurs. Enrollment was discontinued early in the study AZA-MDS-003 due to a higher incidence of early fatal and/or serious adverse reactions in the Onureg arm compared with the placebo arm. Treatment of MDS with Onureg is not recommended outside of controlled trials. Onureg can cause fetal harm when administered to a pregnant woman.

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

FDA Approves Onureg (azacitidine tablets) as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia

A novel pill to treat bleeding from uterine fibroids aims for FDA approval

In continuation of my update on elagolix 

A new drug called elagolix cut blood loss by half over six months in the overwhelming majority of women who participated in two clinical trials published Wednesday in the New England Journal of Medicine.

Elagolix, being developed by AbbVie, was approved by the U.S. Food and Drug Administration in 2018 under the brand name Orilissa to reduce the pain of endometriosis—another common, debilitating female disorder.

The fibroid studies, conducted at 77 sites in the United States and Canada, were led by William D. Schlaff, the chair of obstetrics and gynecology at Thomas Jefferson University in Philadelphia. The results have been submitted to the FDA, which is expected to issue its decision in the first half of 2020.

Elagolix works by suppressing the gonadotropin hormone and the ovarian sex hormones, estrogen and progesterone. In effect, it throws women into a temporary menopause.

Because this suppression can cause bone loss, the researchers gave a subset of women low doses of sex hormones along with elagolix. Of those 395 women, about 70% cut their blood loss by half without suffering more bone thinning than women taking a placebo. The "add-back" hormones also reduced menopausal side effects such as hot flashes and night sweats, although these remained common.

"As a clinician working with patients like this for 40 years, I think this is a valuable clinical tool," Schlaff said of elagolix and add-back hormones. "It's oral, the effect is fast onset, and the side-effect profile is tolerable."

The brand name and pricing that would be used if the FDA approves this use of elagolix have not yet been established for the drug, an AbbVie spokesperson said. The list price for a four-week supply of Orilissa is $907.39.

An estimated 80% of women approaching menopause have fibroids—muscular growths in the uterine wall. Half of those women will develop symptoms, primarily heavy menstrual bleeding, which can lead to a blood iron deficiency. Charlotte Owens, medical director at AbbVie, noted that African American women have a higher risk for uterine fibroids and often develop more severe symptoms than Caucasian women.

After menopause, when the ovaries shut down, menstrual bleeding stops, but many women find the transitional bleeding so troublesome that they seek treatment.

Current options—including drugs that target hormones, procedures that destroy the uterine lining or surgical removal of the fibroids or entire uterus—all have drawbacks. A device called an electric morcellator, which minces fibroids and removes the tissue through tiny incisions, has been largely abandoned because in rare cases it can disseminate an undetected uterine cancer. Philadelphia cardiac surgeon Hooman Noorchashm and his late wife, anesthesiologist Amy Reed, campaigned for a ban on morcellators after her cancer was spread during a hysterectomy.

Existing gonadotropin hormone-suppressing drugs, including one approved in 1989 that Schlaff helped to test in patients, have to be given as injections and take up to two weeks to begin working.

Other companies besides AbbVie have been working to tap the potentially huge market of women with fibroid-related bleeding. A few years ago, Allergan seemed to be in the lead with ulipristal acetate, brand name Esmya, which was already approved in Europe. But after European regulators initiated an investigation into whether the drug led to liver damage in some patients, the FDA declined to approve it.

Myovant Sciences' relugolix, which reduced blood loss in nearly three-quarters of patients in the second of two late-stage clinical trials, is also seeking approval for the drug in combination with add-back hormone medications.

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

FDA Approves Valtoco (diazepam nasal spray) as a Seizure Rescue Treatment

Neurelis, Inc.,  announced that the U.S. Food and Drug Administration (FDA) has approved Valtoco (diazepam nasal spray) as an acute treatment of intermittent, stereotypic episodes of frequent seizure activity (ie, seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in people with epilepsy 6 years of age and older. The unique formulation of Valtoco incorporates Intravail® for consistent and reliable absorption.

“Cluster or acute repetitive seizures are challenging to treat and highly disruptive in the lives of people with epilepsy,” said Neurelis President and CEO Craig Chambliss. “Valtoco was developed to provide an effective combination of reliability, safety and tolerability in a ready-to-use nasal spray. This is a defining moment for Neurelis as Valtoco is our first FDA-approved product. We are excited that we can now offer this treatment option to patients and provide additional support to the epilepsy community.”

Chambliss added that Valtoco was also granted seven years of Orphan Drug Exclusivity by the FDA Office of Orphan Products Development.

Valtoco is a proprietary formulation of diazepam incorporating the Science of Intravail. Intravail transmucosal absorption enhancement technology enables the non-invasive delivery of a broad range of protein, peptide and small molecule drugs. In the United States, there are over 3.4 million people with epilepsy, with approximately 200,000 new patients diagnosed each year. Despite the availability of chronic, daily oral medications to control epilepsy, a significant number of these patients continue to experience seizures. Of these uncontrolled patients, as many as 170,000 are at risk for episodes of frequent seizure activity, also known as cluster or acute repetitive seizures, representing a significant unmet need in the epilepsy community.

“This is an important development in the epilepsy community,” said R. Edward Hogan, MD, Director of the Washington University and Barnes-Jewish Epilepsy Center in St. Louis. “Most seizures that require intervention are treated in an inconvenient manner. To be able to reliably treat seizure activity when and where it happens with a caregiver-administered option like Valtoco is a significant step forward. The availability of Valtoco may positively impact the lives of thousands of people with epilepsy who experience cluster or acute repetitive seizures and their care partners.”

In a long-term, open-label, repeat dose, clinical trial, the safety of Valtoco was evaluated: over 130 patients were enrolled and more than 2,000 seizures were treated. The clinical trial included patients aged 6 and above. “Until recently, approved treatment outside of medical care settings was only available as a rectally administered medication,” Dr. Hogan said. “The FDA approval of diazepam nasal spray is a significant advancement for the epilepsy community.”

Enrique Carrazana, MD, Chief Scientific Officer for Neurelis, notes that Valtoco was generally safe and well tolerated during clinical studies. The most common adverse reactions (at least 4%) were somnolence, headache, and nasal discomfort.

Jacqueline A. French, MD, professor in the Department of Neurology at NYU Langone Health’s Comprehensive Epilepsy Center and Chief Medical & Innovation Officer for the Epilepsy Foundation, commented, “One of the goals of rescue therapy is to treat seizure clusters, recognized as medical emergencies, before negative consequences may be experienced. These consequences may include injury and seizure progression to status epilepticus. Having a seizure rescue treatment that is generally safe, reliable and ready-to-use is very empowering. We encourage all epilepsy patients to work with their doctors to make sure they have a seizure rescue treatment plan in place.”

Indication

Valtoco (diazepam nasal spray) is indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (ie, seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy 6 years of age and older.

https://www.chemdiv.com/valtoco-is-approved-by-fda/