FDA Approves Orserdu (elacestrant) for Patients with ESR1 Mutations in ER+, HER2- Advanced or Metastatic Breast Cancer

The Menarini Group (“Menarini”), a leading Italian pharmaceutical and diagnostics company, announced  the U.S. Food and Drug Administration (FDA)   approval of  Orserdu for the treatment of postmenopausal women or adult men, with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. Stemline Therapeutics (“Stemline”), a wholly-owned subsidiary of the Menarini Group, headquartered in New York and focused on bringing transformational oncology treatments for cancer patients, will commercialize Orserdu in the U.S.

“The FDA approval of Orserdu marks the first ever therapy for ER+, HER2- advanced or metastatic breast cancer patients with ESR1 mutations and we are very proud to offer a targeted therapy addressing this huge unmet need,” commented Elcin Barker Ergun, Chief Executive Officer of the Menarini Group. “We are grateful to the patients, investigators and administrators who participated in the clinical trials that led to this remarkable innovation.”

Orserdu is approved under the FDA’s Priority Review and Fast Track designation based on the results of the registrational Phase III trial EMERALD, that demonstrated statistically significant progression-free survival (PFS) with elacestrant vs SOC endocrine monotherapy (fulvestrant, letrozole, anastrozole, exemestane), meeting both primary endpoints in all patients and in those patients whose tumors harbor ESR1 mutations.

In the group of patients whose tumors had ESR1 mutations, elacestrant reduced the risk of progression or death by 45% (PFS HR=0.55, 95% CI: 0.39, 0.77) vs SOC. A post-hoc analysis of the PFS results based on the duration of prior CDK4/6i inhibitors (CDK4/6i) usage was presented at San Antonio Breast Cancer Symposium (SABCS) in December 2022. The median PFS was 8.6 months on elacestrant vs 1.9 months for SOC, in those patients whose tumors harbored ESR1 mutations and had been treated with a CDK4/6i for at least 12 months.

Safety data is consistent with the other endocrine therapies. Most of the adverse events (AEs), including nausea and musculoskeletal pain were grade 1 and 2. No hematological safety signal was observed and none of the patients in either of the two treatment arms had sinus bradycardia.

“Advanced or metastatic ER+, HER2- breast cancer pre-treated with endocrine-based therapy remains an area of unmet medical need. The last endocrine therapy approved was about 20 years ago, and effective endocrine options for this patient population are needed,” said Dr. Aditya Bardia, MD, MPH, Director of Breast Cancer Research at Mass General Cancer Center, Associate Professor at the Medicine Department at Harvard Medical School, and Principal Investigator for the EMERALD trial. “ESR1 mutations are a known driver of resistance to standard endocrine therapy, and so far, have been difficult to treat. The approval of elacestrant is welcomed as it offers a novel option for patients with ER+, HER2- metastatic breast cancer. This therapy targets the ESR1 mutations in metastatic breast cancer and provides patients with a convenient oral once-daily dose.”

“Each year 300,000 Americans are diagnosed with breast cancer and metastatic breast cancer causes the vast majority of deaths from the disease: more than 43,000 annually. We urgently need new and better treatment options to extend and improve the lives of people with metastatic breast cancer,” said Sonya Negley, Executive Director, Metavivor. “We are thrilled to see the approval of Orserdu, a new oral endocrine therapy, for patients who have tumors that harbor ESR1 mutations, which are present in up to 40% of ER+, HER2- advanced or metastatic breast cancer. We advise patients to get tested for ESR1 mutations at progression in their metastatic treatment, so that their healthcare team can identify the right treatment options for their disease.“

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

FDA Approves Orserdu (elacestrant) for Patients with ESR1 Mutations in ER+, HER2- Advanced or Metastatic Breast Cancer

FDA Approves Jesduvroq (daprodustat) for Anemia Caused by Chronic Kidney Disease for Adults on Dialysis

GSK plc (LSE/NYSE: GSK) announced the US Food and Drug Administration (FDA)  approval of  Jesduvroq (daprodustat), an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), for the once-a-day treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least four months. Jesduvroq is the first innovative medicine for anemia treatment in over 30 years and the only HIF-PHI approved in the US, providing a new oral, convenient option for patients in the US with anemia of CKD on dialysis.

The FDA approval is based on results from the ASCEND-D trial, assessing the efficacy and safety of Jesduvroq for the treatment of anemia of CKD in patients on dialysis. Results were published in the New England Journal of Medicine with additional results published in the New England Journal of Medicine supplementary appendix.

The Safety Information for Jesduvroq includes a boxed warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, and thrombosis of vascular access. Jesduvroq increases the risk of thrombotic vascular events, including major adverse cardiovascular events (MACE). Targeting a hemoglobin level greater than 11 g/dL is expected to further increase the risk of death and arterial venous thrombotic events, as occurs with erythropoietin stimulating agents (ESAs), which also increase erythropoietin levels. No trial has identified a hemoglobin target level, dose of Jesduvroq, or dosing strategy that does not increase these risks. Use the lowest dose of Jesduvroq sufficient to reduce the need for red blood cell transfusions. Jesduvroq has not been shown to improve quality of life, fatigue, or patient well-being. Jesduvroq is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia or for treatment of anemia of chronic kidney disease in patients who are not on dialysis.

CKD is an increasing global health burden affecting 700 million patients worldwide, with an estimated one in seven patients also developing anemia.1,2 When left untreated or undertreated, anemia of CKD is associated with poor clinical outcomes and leads to a substantial burden on patients and healthcare systems.3 There is an unmet need for oral treatment options with efficacy and safety comparable to current treatments.

LaVarne Burton, President and Chief Executive Officer, American Kidney Fund, said: “Anemia of CKD can be a debilitating condition that is challenging to manage. This news means that patients on dialysis who are living with anemia of CKD now have another treatment option to help manage their anemia.”

A marketing authorisation application for daprodustat is currently under review with the European Medicines Agency, with a regulatory decision anticipated in the first half of 2023. In June 2020, daprodustat tablets were approved by Japan’s Ministry of Health, Labour and Welfare for the treatment of patients with anemia of CKD. In Japan, the brand name for daprodustat is Duvroq, where it is the market leader and preferred HIF-PHI.

Tony Wood, President and Chief Scientific Officer, GSK, said: “Over the last several decades, there has been little innovation in anemia of CKD. We are proud to have developed Jesduvroq as a new oral treatment where there is a patient desire for more options.”

The FDA approval is based on results from the ASCEND-D trial, assessing the efficacy and safety of Jesduvroq for the treatment of anemia of CKD in patients on dialysis. Results were published in the New England Journal of Medicine with additional results published in the New England Journal of Medicine supplementary appendix.

The Safety Information for Jesduvroq includes a boxed warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, and thrombosis of vascular access. Jesduvroq increases the risk of thrombotic vascular events, including major adverse cardiovascular events (MACE). Targeting a hemoglobin level greater than 11 g/dL is expected to further increase the risk of death and arterial venous thrombotic events, as occurs with erythropoietin stimulating agents (ESAs), which also increase erythropoietin levels. No trial has identified a hemoglobin target level, dose of Jesduvroq, or dosing strategy that does not increase these risks. Use the lowest dose of Jesduvroq sufficient to reduce the need for red blood cell transfusions. Jesduvroq has not been shown to improve quality of life, fatigue, or patient well-being. Jesduvroq is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia or for treatment of anemia of chronic kidney disease in patients who are not on dialysis.

CKD is an increasing global health burden affecting 700 million patients worldwide, with an estimated one in seven patients also developing anemia.1,2 When left untreated or undertreated, anemia of CKD is associated with poor clinical outcomes and leads to a substantial burden on patients and healthcare systems.3 There is an unmet need for oral treatment options with efficacy and safety comparable to current treatments.

LaVarne Burton, President and Chief Executive Officer, American Kidney Fund, said: “Anemia of CKD can be a debilitating condition that is challenging to manage. This news means that patients on dialysis who are living with anemia of CKD now have another treatment option to help manage their anemia.”

A marketing authorisation application for daprodustat is currently under review with the European Medicines Agency, with a regulatory decision anticipated in the first half of 2023. In June 2020, daprodustat tablets were approved by Japan’s Ministry of Health, Labour and Welfare for the treatment of patients with anemia of CKD. In Japan, the brand name for daprodustat is Duvroq, where it is the market leader and preferred HIF-PHI.

The FDA approval is based on results from the ASCEND-D trial, assessing the efficacy and safety of Jesduvroq for the treatment of anemia of CKD in patients on dialysis. Results were published in the New England Journal of Medicine with additional results published in the New England Journal of Medicine supplementary appendix.

The Safety Information for Jesduvroq includes a boxed warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, and thrombosis of vascular access. Jesduvroq increases the risk of thrombotic vascular events, including major adverse cardiovascular events (MACE). Targeting a hemoglobin level greater than 11 g/dL is expected to further increase the risk of death and arterial venous thrombotic events, as occurs with erythropoietin stimulating agents (ESAs), which also increase erythropoietin levels. No trial has identified a hemoglobin target level, dose of Jesduvroq, or dosing strategy that does not increase these risks. Use the lowest dose of Jesduvroq sufficient to reduce the need for red blood cell transfusions. Jesduvroq has not been shown to improve quality of life, fatigue, or patient well-being. Jesduvroq is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia or for treatment of anemia of chronic kidney disease in patients who are not on dialysis.

CKD is an increasing global health burden affecting 700 million patients worldwide, with an estimated one in seven patients also developing anemia.1,2 When left untreated or undertreated, anemia of CKD is associated with poor clinical outcomes and leads to a substantial burden on patients and healthcare systems.3 There is an unmet need for oral treatment options with efficacy and safety comparable to current treatments.

LaVarne Burton, President and Chief Executive Officer, American Kidney Fund, said: “Anemia of CKD can be a debilitating condition that is challenging to manage. This news means that patients on dialysis who are living with anemia of CKD now have another treatment option to help manage their anemia.”

A marketing authorisation application for daprodustat is currently under review with the European Medicines Agency, with a regulatory decision anticipated in the first half of 2023. In June 2020, daprodustat tablets were approved by Japan’s Ministry of Health, Labour and Welfare for the treatment of patients with anemia of CKD. In Japan, the brand name for daprodustat is Duvroq, where it is the market leader and preferred HIF-PHI.

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

https://go.drugbank.com/drugs/DB11682

FDA Approves Jesduvroq (daprodustat) for Anemia Caused by Chronic Kidney Disease for Adults on Dialysis

FDA Approves Miebo (perfluorohexyloctane) Ophthalmic Solution for the Treatment of the Signs and Symptoms of Dry Eye Disease

Bausch + Lomb Corporation, announced that the U.S. Food and Drug Administration (FDA) has approved Miebo™ (perfluorohexyloctane ophthalmic solution; formerly known as NOV03), for the treatment of the signs and symptoms of dry eye disease (DED). Miebo is the first and only FDA-approved treatment for DED that directly targets tear evaporation.

“Today’s FDA approval of Miebo further advances DED treatment by addressing a significant unmet need for millions of people suffering with this disease,” said Brent Saunders, chairman and CEO, Bausch + Lomb. “We are proud to bring to market the first and only prescription eye drop approved in the United States for the treatment of DED that directly targets evaporation. We expect to make Miebo commercially available in the second half of this year.”

DED affects millions of Americans and is one of the most common ocular surface disorders.1 A leading cause of DED is excessive tear evaporation, which due to an altered tear lipid layer, is often associated with the clinical signs of Meibomian gland dysfunction (MGD). An unstable tear film triggers increased ocular surface desiccation, inflammation and damage to the ocular surface.2,3 Miebo is designed to reduce tear evaporation at the ocular surface.4,5

In GOBI and MOJAVE, two phase 3 pivotal clinical trials which enrolled more than 1,200 patients (randomized 1:1 to Miebo or hypotonic saline) with a history of DED and clinical signs of MGD, Miebo consistently met its primary clinical sign and patient-reported symptom endpoint.

"In the two pivotal clinical trials, Miebo addressed the persistent and chronic nature of DED by providing sustained improvement in both the signs and symptoms of DED,” said Preeya Gupta, M.D., cornea and cataract surgeon, Triangle Eye Consultants, Raleigh, North Carolina. “Because Miebo inhibits evaporation, it may be an appropriate treatment option for patients whose tear evaporation exceeds tear supply.”

“Tear evaporation, which is a leading driver of DED, presents a significant treatment challenge. With the approval of Miebo, eye care professionals can now take a new approach to DED therapy with a first-in-class water- and preservative-free prescription treatment option that specifically addresses tear evaporation,” said Paul Karpecki, O.D., director, Cornea and External Disease, Kentucky Eye Institute, and associate professor, University of Pikeville, Kentucky College of Optometry.

Ref : Read More  

FDA Approves Lumryz (sodium oxybate) for Cataplexy or Excessive Daytime Sleepiness in Adults with Narcolepsy

In continuation of my update on Lumryz

Avadel Pharmaceuticals plc   a biopharmaceutical company announced   the U.S. Food & Drug Administration (FDA)  final approval to Lumryz, an extended-release formulation of sodium oxybate indicated to be taken once at bedtime for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. With final approval, Lumryz becomes the first and only FDA approved once-at-bedtime oxybate for people living with narcolepsy. Lumryz was additionally granted Orphan Drug Exclusivity by the FDA.

“Today’s landmark approval and receipt of Orphan Drug Exclusivity represents a major milestone for both Avadel and people living with narcolepsy. As we have heard from key stakeholders, previously approved narcolepsy therapies have the potential to disrupt sleep by either causing insomnia or through forced awakening during the middle of the night for their crucial second dose. Lumryz can now offer people with narcolepsy the opportunity for an uninterrupted night sleep while receiving the full benefit of their prescribed treatment in one single bedtime dose that addresses their symptoms of narcolepsy,” said Greg Divis, Chief Executive Officer of Avadel. “We would like to thank the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have tirelessly partnered with us throughout the drug development process and look forward to providing the narcolepsy community access to now approved Lumryz.”

Narcolepsy is a chronic neurological condition that impairs the brain's ability to regulate the sleep-wake cycle. The condition affects approximately one in 2,000 people in the United States with the cardinal symptom of EDS. Additional symptoms can vary by person but may include disrupted nighttime sleep, a sudden loss of muscle tone usually triggered by strong emotion (cataplexy), sleep paralysis and hallucinations.

“This long-awaited therapy for people living with narcolepsy fills a critical unmet need by avoiding the burden of a second middle-of-the-night dose that immediate-release oxybate products require. The once-at-bedtime dosing regimen of Lumryz may help restore a more natural sleep-wake cycle,” said Michael J. Thorpy, M.D., an investigator from the REST-ON Phase 3 trial and Director at the Sleep-Wake Disorders Center at Montefiore Medical Center and Professor of Neurology at the Albert Einstein College of Medicine.

The FDA’s final approval of Lumryz was based on positive results from the pivotal Phase 3 REST-ON clinical study completed in March 2020. In the REST-ON Phase 3 trial, once-at-bedtime Lumryz demonstrated highly statistically significant (p<0.001) and clinically meaningful improvement compared to placebo across all three co-primary endpoints (Maintenance of Wakefulness Test, Clinical Global Impression-Improvement and mean weekly cataplexy attacks) for all three doses evaluated, 6, 7.5 and 9 grams.

With this approval, the FDA has also found Lumryz to be clinically superior to currently marketed twice-nightly oxybate products and granted Lumryz seven years of Orphan Drug Exclusivity. In particular, FDA found that Lumryz makes a major contribution to patient care over currently available, twice-nightly oxybate products by providing a once-nightly dosing regimen that avoids nocturnal arousal to take a second dose. The FDA's Orphan Drug program is designed to support the development of drugs that treat a condition affecting less than 200,000 U.S. patients. The seven-year market exclusivity for Lumryz began on the date of FDA approval, May 1, 2023.

“For people living with narcolepsy, and for all of us who advocate for this community, the approval of Lumryz is an important step forward,” said Julie Flygare, JD, President and CEO of Project Sleep. “People living with narcolepsy will finally have a new treatment option to manage EDS and cataplexy, and the fact that this new oxybate option allows for reduced dosing frequency is a game-changing advancement that shows Avadel’s commitment to understanding the patient experience. We look forward to continued collaboration with Avadel as part of a shared mission to positively impact the lives of people with narcolepsy.”

Lumryz has a boxed warning as a central nervous system depressant, and for its potential for abuse and misuse. Lumryz is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the Lumryz REMS. Most common adverse reactions (incidence > 5% and greater than placebo) reported for all doses of Lumryz combined were nausea, dizziness, enuresis, headache, and vomiting.

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

FDA Approves Lumryz (sodium oxybate) for Cataplexy or Excessive Daytime Sleepiness in Adults with Narcolepsy

FDA Approves Mydcombi (tropicamide and phenylephrine hydrochloride) Ophthalmic Spray for Inducing Mydriasis

In continuation of my update on phenylephrine hydrochloride

Phenylephrine

Tropicamide

Eyenovia, Inc, announced  the U.S. Food and Drug Administration (FDA)  approval of Mydcombi (tropicamide and phenylephrine hydrochloride ophthalmic spray) 1%/2.5% for inducing mydriasis for diagnostic procedures and in conditions where short term pupil dilation is desired. This represents the first approved fixed dose combination of tropicamide and phenylephrine in the United States and also the first product using Eyenovia’s proprietary Optejet device to be approved by any regulatory authority.

Mydcombi is designed to improve the efficiency of the estimated 106 million office-based comprehensive eye exams performed every year in the United States, as well as the estimated 4 million pharmacologic mydriasis applications for cataract surgery. The product is contraindicated and should not be used in patients with known hypersensitivity to any component of the formulation.

“The approval of Mydcombi, our first FDA approved product, represents the culmination of years of tireless effort by the entire Eyenovia team, and I would like to express my sincere gratitude to the associates and technical experts who helped advance this important program through this transformational milestone,” stated Michael Rowe, chief executive officer of Eyenovia. “We look forward to introducing Mydcombi to key offices beginning this summer while we bring our internal manufacturing capabilities on-line for 2024.”

“Perhaps more importantly, FDA approval of Mydcombi provides critical validation of the Optejet as it is the first product approved using the Optejet platform, which is core not only to our internal development programs, including MicroLine for presbyopia, but our partnered programs as well. We see opportunities to unlock significant opportunities in the future treatment of other ophthalmic conditions including glaucoma and dry eye. I am confident in our ability to maintain our current momentum.”

“I am proud of our team for this significant achievement – which represents many ‘firsts’ for eye care,” stated Dr. Sean Ianchulev, Founder and Chairman of Eyenovia’s Board of Directors. “The use of eye dropper bottles has presented challenges for dosing in ophthalmologic settings in millions of patients. We can do better now using sophisticated micro-array print delivery with physiologic dosing that is similar to the natural tear film volume.”

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

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

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FDA Grants Accelerated Approval for Qalsody (tofersen) for the Treatment of Amyotrophic Lateral Sclerosis Associated with a Mutation in the SOD1 Gene

Biogen Inc. (Nasdaq: BIIB) announced the U.S. Food and Drug Administration (FDA)   approval of  Qalsody (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with Qalsody. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).1 The ongoing Phase 3 ATLAS study of tofersen in people with presymptomatic SOD1-ALS will serve as the confirmatory trial.

Neurofilaments are proteins that are released from neurons when they are damaged, making them a marker of neurodegeneration.6

“For more than a decade, Biogen has been steadfast in our commitment to pursuing treatments for ALS, and I want to thank the scientists as well as the entire ALS community who have all worked tirelessly to bring this first-of-its-kind treatment to people with SOD1-ALS,” said Christopher A. Viehbacher, President and Chief Executive Officer of Biogen. “Today also marks a pivotal moment in ALS research as we gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS. We believe this important scientific advancement will further accelerate innovative drug development for ALS.”

Qalsody is the first approved treatment to target a genetic cause of ALS. Biogen collaborated with Ionis Pharmaceuticals on the early development of tofersen.

Warnings and precautions associated with Qalsody were serious neurologic events, including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis. If symptoms consistent with myelitis, radiculitis papilledema, elevated intracranial, or aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care. Management may require interruption or discontinuation of Qalsody. The most common adverse reactions that occurred in ≥10% of Qalsody treated participants and more than the placebo arm were pain, fatigue, arthralgia, cerebrospinal (CSF) white blood cell increased, and myalgia.

“Since SOD1 mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments. Qalsody offers families who have lost generation after generation in the prime of their life to this devastating disease a therapy targeting the underlying cause of SOD1-ALS. Today marks an important moment in ALS research as Qalsody is the first ALS treatment approved based on a biomarker,” said Jean Swidler, chair of Genetic ALS & FTD: End the Legacy. “We are excited to see what future therapies are developed now that it is understood that lowering levels of neurofilament provides important evidence that a treatment is affecting the neurodegenerative process.”

The efficacy of Qalsody was assessed in a 28-week randomized, double-blind, placebo-controlled clinical study in patients 23 to 78 years of age with weakness attributable to ALS and a SOD1 mutation confirmed by a central laboratory. One hundred eight (108) patients were randomized 2:1 to receive treatment with either Qalsody 100 mg (n=72) or placebo (n=36) for 24 weeks (3 loading doses followed by 5 maintenance doses). Concomitant riluzole and/or edaravone use was permitted for patients and at baseline 62% of patients were taking riluzone, and 8% of patients were taking edaravone.

Over 28 weeks in VALOR, participants in the primary analysis population (n=60) treated with Qalsody experienced less decline from baseline as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) compared to placebo, though the results were not statistically significant (Qalsody-placebo adjusted mean difference [95% CI]: 1.2 [-3.2, 5.5]). In the overall intent-to-treat population (n=108), Qalsody-treated participants experienced a 55% reduction in plasma NfL compared to a 12% increase in placebo-treated participants (difference in geometric mean ratios for Qalsody to placebo: 60%; nominal p<0.0001). Additionally, levels of CSF SOD1 protein, an indirect measure of target engagement, were reduced by 35% in the Qalsody-treated group compared to 2% in the corresponding placebo group (difference in geometric mean ratios for Qalsody to placebo: 34%; nominal p<0.0001).

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

https://go.drugbank.com/drugs/DB14782

FDA Grants Accelerated Approval for Qalsody (tofersen) for the Treatment of Amyotrophic Lateral Sclerosis Associated with a Mutation in the SOD1 Gene

FDA Approves Abilify Asimtufii (aripiprazole) for the Treatment of Schizophrenia or Maintenance Monotherapy Treatment of Bipolar I Disorder in Adults

In continuation of my update on aripiprazole

Otsuka Pharmaceutical Co., Ltd. (Otsuka) and Lundbeck Pharmaceuticals LLC (Lundbeck) announced the U.S. Food and Drug Administration (FDA)   approval of  the New Drug Application (NDA) for Abilify Asimtufii® (aripiprazole) extended-release injectable suspension for intramuscular use, a once-every-two-months injection for the treatment of schizophrenia in adults or for maintenance monotherapy treatment of bipolar I disorder in adults.

Abilify Asimtufii offers two months of sustained therapeutic concentrations with one dose. Each dose is provided in a single-chamber, prefilled syringe, and is administered by a healthcare professional to appropriate patients via intramuscular injection in the gluteal muscle. Long-acting injectables provide continuous delivery of antipsychotic medication and can maintain therapeutic plasma concentrations, which may help to maintain symptom control of schizophrenia and bipolar I disorder.

"We are pleased to offer this new treatment option for people living with schizophrenia or bipolar I disorder that may delay the time to relapse," said John Kraus, M.D., Ph.D., executive vice president and chief medical officer at Otsuka Pharmaceutical Development & Commercialization, Inc. "This approval underscores Otsuka's commitment to innovate and continuously evolve to meet the needs of the communities we serve."

The efficacy of Abilify Asimtufii is based on the adequate and well-controlled studies of Abilify Maintena (aripiprazole) in the treatment of schizophrenia or maintenance treatment of bipolar I disorder in adults. The aripiprazole concentrations of Abilify Asimtufii were explored in a pharmacokinetic bridging study which was a 32-week, open-label, multiple-dose, randomized, parallel-arm, multicenter study (N=266) in patients living with schizophrenia and bipolar I disorder. The once-every-two-months, long-acting injectable formulation in 960 mg and 720 mg prefilled syringes delivers sustained plasma concentrations similar to that demonstrated in studies with aripiprazole monohydrate once-monthly, long-acting injectable, resulting in similar sustained efficacy.

"This approval is important news for patients, families, and healthcare providers. We hope that the use of Abilify Asimtufii in treatment plans will have a positive impact on those living with schizophrenia or bipolar I disorder," said Johan Luthman, executive vice president, R&D, Lundbeck. "We are grateful to the patients and researchers who made this major milestone possible."

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

FDA Approves Abilify Asimtufii (aripiprazole) for the Treatment of Schizophrenia or Maintenance Monotherapy Treatment of Bipolar I Disorder in Adults

FDA Approves Uzedy (risperidone) Extended-Release Injectable Suspension for the Treatment of Schizophrenia in Adults

In continuation of my update on Uzedy (risperidone)

Teva Pharmaceuticals,  announced  the U.S. Food and Drug Administration (FDA)  approval of  Uzedy (risperidone) extended-release injectable suspension for the treatment of schizophrenia in adults. Uzedy is the first subcutaneous, long-acting formulation of risperidone that utilizes SteadyTeq™, a copolymer technology proprietary to MedinCell that controls the steady release of risperidone. Therapeutic blood concentrations are reached within 6-24 hours of a single dose. 

“Uzedy embodies Teva’s commitment to bringing innovative advances to patients and to providing people living with schizophrenia an important new treatment option that was designed to address certain treatment challenges and may decrease the risk of relapse,” said Richard Francis, President and CEO of Teva. “The approval of Uzedy is a culmination of a multidisciplinary effort across Teva and MedinCell to bring this important treatment to market. This milestone is a testament to advancing our robust biopharmaceutical pipeline of innovative medicines that aim to support more people living with mental health disorders and neurological diseases in the coming years.”

Approximately 80% of patients with schizophrenia experience multiple relapses over the first five years of treatment,2 most commonly due to suboptimal adherence to treatment with oral antipsychotics. Each relapse carries a biological risk of loss of function, treatment refractoriness, and changes in brain morphology.3,4

Schizophrenia is a chronic, progressive and severely debilitating mental health disorder that affects how one thinks, feels and acts.5 This approval is based on data from two Phase 3 trials evaluating Uzedy in patients with schizophrenia: TV46000-CNS-30072 (the RISE Study – The Risperidone Subcutaneous Extended-Release Study) and TV46000-CNS-30078 (the SHINE Study – A Study to Test TV-46000 for Maintenance Treatment of Schizophrenia).

“The approval of the first product formulated with our technology is a pivotal moment for MedinCell and for the many patients who will benefit,” said Christophe Douat, CEO of MedinCell. “We are committed to supporting patients through innovative therapy options. It continues to be a wonderful journey with Teva, an ideal partner to harness the full potential of Uzedy. Our technology reaching commercial stage marks the start of an exciting new era for MedinCell and we are extremely proud to share this very special moment with all our employees and shareholders.”

The use of novel SteadyTeq technology in Uzedy controls the release of risperidone over time. The initiation of treatment requires no loading dose or oral supplementation. Therapeutic blood concentrations are reached within 6-24 hours of a single dose. 

“Treatments for schizophrenia are largely prescribed as daily oral medications, which can present challenges with adherence due to missed doses. Lack of adherence to treatment with oral antipsychotics is the most common cause of relapse in schizophrenia  so there’s a role for therapies that are dosed in one- or two-month dosing intervals to help prevent relapse,” said Christoph Correll, MD, professor of psychiatry at the Zucker School of Medicine, Hempstead, NY. “As a clinician, I am excited to now have a new treatment option that reduces the risk of relapse1 for this complex disease and helps address some of the barriers around receiving schizophrenia treatment.”

https://pubchem.ncbi.nlm.nih.gov/compound/Risperidone#section=2D-Structure

FDA Approves Uzedy (risperidone) Extended-Release Injectable Suspension for the Treatment of Schizophrenia in Adults

FDA Grants Accelerated Approval to Filspari (sparsentan) for the Reduction of Proteinuria in IgA Nephropathy

Travere Therapeutics, Inc. (Nasdaq: TVTX) announced  the U.S. Food and Drug Administration (FDA)  approval to Filspari™ (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g.

This indication is granted under accelerated approval based on reduction in proteinuria. It has not been established whether Filspari slows kidney function decline in patients with IgAN. The continued approval of Filspari may be contingent upon confirmation of a clinical benefit in the ongoing Phase 3 PROTECT Study, which is designed to demonstrate whether Filspari slows kidney function decline. Topline results from the two-year confirmatory endpoints in the PROTECT Study are expected in the fourth quarter of 2023 and are intended to support traditional approval of Filspari.

Filspari, a once-daily oral medication is designed to selectively target two critical pathways in the disease progression of IgAN (endothelin-1 and angiotensin II), and is the first and only non-immunosuppressive therapy approved for the treatment of this condition. IgAN is a rare kidney disease (RKD) and a leading cause of kidney failure due to glomerular disease, affecting up to 150,000 people in the U.S., with approximately 30,000 to 50,000 of such patients estimated to be addressable under the indication approved via accelerated approval. The Company expects Filspari to be available beginning the week of February 27, 2023, and will be providing a comprehensive patient support program throughout the patient’s treatment journey.

“The accelerated approval of Filspari is a significant milestone on our path to advancing a transformative treatment for the IgA nephropathy community,” said Eric Dube, Ph.D., president and chief executive officer, Travere Therapeutics. “As a first-of-its-kind, non-immunosuppressive therapy, we believe Filspari has the potential to ultimately become the new standard of care for IgA nephropathy and offer hope to those living with this condition who until now have had few treatment options. We are grateful to the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have worked alongside us to develop this innovative first-in-class therapy.”

“Today’s approval of Filspari sets the stage for a new standard of care for IgA nephropathy patients. A high proportion of individuals diagnosed with this disease do not sufficiently respond to the historical standard treatment, which has been therapies that are not indicated for IgA nephropathy. These treatments include hypertension drugs such as angiotensin-receptor blockers (ARBs), angiotensin-converting enzyme (ACE) inhibitors and systemic glucocorticoids. As a result, many patients have struggled to manage their disease and have progressed more quickly to kidney failure,” said Dr. Brad Rovin, MD, Medical Director at Ohio State University Center for Clinical Research Management, Director of the Division for Nephrology, and steering committee member for the PROTECT clinical trial. “The approval of this innovative treatment is founded in data from the largest head-to-head Phase 3 clinical trial in IgA nephropathy. It is exciting to see that adult patients who are at risk of rapid disease progression, many of whom have waited a very long time for a treatment like this, now have hope for a better future.”

The approval of Filspari, granted under the FDA’s accelerated approval pathway, is based on clinically meaningful and statistically significant improvements in proteinuria compared to an active comparator in the pivotal and ongoing Phase 3 PROTECT Study, the largest head-to-head interventional study to date in IgAN. The PROTECT Study is a global, randomized, multicenter, double-blind, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of Filspari, compared to 300 mg of irbesartan, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite maximal tolerated ACE or ARB therapy.  

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

FDA Grants Accelerated Approval to Filspari(sparsentan) for the Reduction of Proteinuria in IgA Nephropathy

FDA Approves Veozah (fezolinetant) for the Treatment of Vasomotor Symptoms Due to Menopause

Astellas Pharma Inc. announced  the U.S. Food and Drug Administration (FDA)   approval of  Veozah (fezolinetant) 45 mg once daily for the treatment of moderate to severe vasomotor symptoms (VMS) due to menopause  on May 12. Veozah is the first nonhormonal neurokinin 3 (NK3) receptor antagonist approved to treat VMS due to menopause.

“Today’s approval of fezolinetant is a significant and, I believe, long awaited milestone for individuals in the U.S. who experience moderate to severe vasomotor symptoms during the menopausal transition,” said Genevieve Neal-Perry, M.D., Ph.D., Chair, UNC School of Medicine Department of Obstetrics and Gynecology. “This therapy is based on our understanding of the biology behind hot flashes. I’m excited to know that patients will have the option to choose this nonhormonal treatment.”

Before menopause, there is a balance between estrogens (hormones made by a woman’s ovaries) and neurokinin B (NKB), a brain chemical. This balance regulates the body’s temperature control center located in a specific area of the brain. As the body goes through menopause, estrogens decline and this balance is disrupted. This imbalance can lead to very uncomfortable symptoms called VMS. Veozah helps to restore the balance by blocking NKB in the temperature control center to reduce the number and intensity of hot flashes.

“Veozah uses a novel mechanism of action to target the root cause of VMS due to menopause,” said Marci English, Vice President and Head of BioPharma Development, Astellas. “FDA approval of this new treatment for moderate to severe VMS due to menopause is a testament to Astellas’ commitment to delivering innovative therapies in areas of unmet need that have been underserved, including women’s health.”

The approval is supported by results from the BRIGHT SKY™ program, which included three Phase 3 clinical trials as part of a development program that collectively enrolled over 3,000 individuals across the U.S., Canada and Europe. Results from the SKYLIGHT 1™ and SKYLIGHT 2™ pivotal trials characterize the efficacy and safety of fezolinetant for the treatment of moderate to severe VMS due to menopause. Data from the SKYLIGHT 4™ safety study further characterizes the long-term safety profile of fezolinetant.

VMS, characterized by hot flashes and/or night sweats, are common symptoms of menopause. VMS are the most common symptoms of menopause for which women seek treatment. In the U.S., about 60% to 80% of women experience these symptoms during or after the menopausal transition. VMS can have a disruptive impact on women’s daily activities and overall quality of life.

“Today’s approval of fezolinetant is a significant and, I believe, long awaited milestone for individuals in the U.S. who experience moderate to severe vasomotor symptoms during the menopausal transition,” said Genevieve Neal-Perry, M.D., Ph.D., Chair, UNC School of Medicine Department of Obstetrics and Gynecology. “This therapy is based on our understanding of the biology behind hot flashes. I’m excited to know that patients will have the option to choose this nonhormonal treatment.”

Before menopause, there is a balance between estrogens (hormones made by a woman’s ovaries) and neurokinin B (NKB), a brain chemical. This balance regulates the body’s temperature control center located in a specific area of the brain. As the body goes through menopause, estrogens decline and this balance is disrupted. This imbalance can lead to very uncomfortable symptoms called VMS. Veozah helps to restore the balance by blocking NKB in the temperature control center to reduce the number and intensity of hot flashes.

“Veozah uses a novel mechanism of action to target the root cause of VMS due to menopause,” said Marci English, Vice President and Head of BioPharma Development, Astellas. “FDA approval of this new treatment for moderate to severe VMS due to menopause is a testament to Astellas’ commitment to delivering innovative therapies in areas of unmet need that have been underserved, including women’s health.”

The approval is supported by results from the BRIGHT SKY™ program, which included three Phase 3 clinical trials as part of a development program that collectively enrolled over 3,000 individuals across the U.S., Canada and Europe. Results from the SKYLIGHT 1™ and SKYLIGHT 2™ pivotal trials characterize the efficacy and safety of fezolinetant for the treatment of moderate to severe VMS due to menopause. Data from the SKYLIGHT 4™ safety study further characterizes the long-term safety profile of fezolinetant.

Ref : https://en.wikipedia.org/wiki/Fezolinetant

FDA Approves Veozah (fezolinetant) for the Treatment of Vasomotor Symptoms Due to Menopause

FDA Approves RizaFilm (rizatriptan) Oral Film for the Treatment of Acute Migraine

In continuation of my update on rizatriptan

IntelGenx Corp. (TSX:IGX)(OTCQB:IGXT),  announced the U.S. Food and Drug Administration  approval of RizaFilm® VersaFilm® 505(b)(2) new drug application (NDA) for the treatment of acute migraine.

RizaFilm® is a proprietary oral thin film formulation of rizatriptan benzoate, the active ingredient in Merck & Co.'s Maxalt®. The global migraine drugs market was valued at nearly $3 billion in 2021 and is expected to reach nearly $11 billion by 2030, representing a compound annual growth rate of 15.6%.

In December 2018, IntelGenx entered into a definitive licensing, development and supply agreement with Gensco® Pharma (“Gensco”) for the exclusive commercialization of RizaFilm® in the United States. Under the terms of the agreement, IntelGenx is entitled to receive royalty payments based on net profits of RizaFilm®; and is eligible to receive pre-specified payments upon the achievement of certain regulatory and commercial milestones.

“Following a successful pre-approval inspection by the FDA of our Montreal manufacturing facility earlier this month, we are thrilled to reach this milestone and excited to soon introduce what will be the first oral thin film for the treatment of acute migraines available in the U.S.,” said Andre Godin, IntelGenx’s President and CFO. “According to the American Migraine Foundation, 39 million or 12% of Americans suffer from migraine, which is the second leading cause of disability nationwide. We are looking forward to working with our commercialization partner, Gensco, to bring this innovative migraine therapeutic to patients seeking convenient administration and quick relief from their pain. In addition to these benefits, RizaFilm® is well suited to the approximately 80% of patients who have migraine-related nausea3, as well as those who have difficulty swallowing.”

Rea More..         

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

FDA Approves RizaFilm (rizatriptan) Oral Film for the Treatment of Acute Migraine

FDA Approves Joenja (leniolisib) for the Treatment of Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announced the USFDA  approval of Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja® application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja® is expected to launch in the US in early April and Dr. Eveline Wu, MD, MSCR, Division Chief, Paediatric Rheumatology & Associate Professor of Paediatric Rheumatology and Allergy/Immunology at The University of North Carolina School of Medicine, said:

"The FDA approval of Joenja® is an exciting moment for the APDS community and offers to transform the treatment pathway for patients and families affected by this rare disease. This approval means that they will, for the first time, have access to an approved treatment, which has the potential to change the standard of care for the patient population suffering from APDS."

Vicki Modell, co-founder of the Jeffrey Modell Foundation, an international, non-profit, organization dedicated to helping individuals and family members affected by primary immunodeficiency disorders, commented:

"The approval of Pharming's Joenja® is an important step toward making a difference in the lives of individuals living with APDS who experience severe, life-altering and progressive symptoms. The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community. The Jeffrey Modell Foundation's mission of hope, advocacy and action is dedicated to early diagnosis, genetic sequencing, treatments and ultimately, future cures for primary immunodeficiencies."

Sijmen de Vries, Chief Executive Officer of Pharming, commented:

"This FDA approval of Joenja® is an important milestone for people living with APDS who will now have access to the first approved treatment option specifically for this debilitating disease. Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS. We are grateful to the patients, caregivers, and physicians who participated in the clinical trials who have made today's approval a reality. I would also like to thank the Pharming and the Novartis teams who have supported the development of Joenja® and can, therefore, be justifiably proud of this FDA approval.

Today also marks a landmark event for Pharming and demonstrates our commitment to transforming the lives of patients who suffer from rare diseases. The approval and near-term launch of Joenja®, our second commercial product, brings us closer to our goal of becoming a leading global rare disease company dedicated to patient communities with unmet medical needs."

APDS is a rare primary immunodeficiency that was first characterized in 2013 and is currently estimated to affect 1 to 2 people per million. It is caused by genetic variants in either one of two identified genes, known as PIK3CD or PIK3R1, which are vital to the normal development and function of immune cells in the body. While people with APDS may suffer from a wide variety of symptoms, the most common are frequent and severe infections of the ears, sinuses, and upper and lower respiratory tracts. Infections usually begin in infancy. People with APDS are susceptible to swollen lymph nodes or an enlarged spleen (splenomegaly), as well as autoimmunity and inflammatory symptoms. People with APDS may also be at higher risk for cancers like lymphoma.

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

FDA Approves Joenja (leniolisib) for the Treatment of Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)