Drugs
The University of Illinois Chicago College of Pharmacy is petitioning FDA to revoke the agency order stopping the U.S. Pharmacopeia (USP) from creating modified monographs of biological drugs. The petition says FDA could reduce the cost of developing biosimilars without compromising their safety and efficacy by removing the side-
Drugs
FDA has removed a partial clinical hold on Stoke Therapeutics’ zorevunersen (STK-001) and its use in children and adolescents with Dravet syndrome. The therapy was under a modified partial hold since 3/2023 that allowed the administration of a single dose of STK-001 (70 mg) in its ongoing
Drugs
FDA has given BioVie approval to begin its Phase 2 SUNRISE-PD trial in Parkinson’s disease patients. The company says the agency reviewed its clinical trial and provided a single, actionable comment recommending the inclusion of the Part II score from the Movement Disorder Society’s Unified Parkinson’s
Drugs
FDA has granted Kyverna Therapeutics a regenerative medicine advanced therapy designation for the company’s autologous CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, and its use to treat patients suffering from progressive myasthenia gravis. The autoimmune disorder is associated with muscle weakness in tissues throughout the
Drugs
Lykos Therapeutics says it will pursue FDA’s dispute resolution process after the agency issued an 8/9 complete response letter on its NDA for midomafetamine (MDMA) capsules for a proposed indication of treating post-traumatic stress disorder (PTSD). The agency’s Psychopharmacologic Drugs Advisory Committee (PDAC) voted in June
Drugs
FDA has approved an Ascendis Pharma NDA for Yorvipath (palopegteriparatide) for treating hypoparathyroidism in adults. The rare endocrine disease is caused by insufficient levels of parathyroid hormone that can impact multiple organs. Approval was based on data from the global Phase 2 PaTH Forward and Phase 3 PaTHway trials, according
Drugs
FDA has approved an ARS Pharmaceuticals NDA for neffy (epinephrine nasal spray) for the emergency treatment of allergic reactions (Type I), including anaphylaxis, in adult and pediatric patients. “Today's approval provides the first epinephrine product for the treatment of anaphylaxis that is not administered by injection,” CDER Division
Drugs
Braun Medical has recalled (Class 1) two lots of 0.9% sodium chloride for injection USP 1,000 mL due to the potential for particulate matter in the drug container and fluid leakage. “The affected batches were inadvertently released to the market prior to the completion of the required acceptance
Drugs
FDA has lifted a clinical hold against 4D Molecular Therapeutics’ 4D-310 therapy being studied for treating Fabry disease cardiomyopathy, and the company says enrollment in the Phase 1/2 INGLAXA study will resume in the coming months. The 2/2023 clinical hold was ordered after the company reported three
Drugs
FDA has released the form FDA-483 issued following a 3/11-4/3 inspection at Imprimis NJOF, LLC, a Ledgewood, NJ, outsourcing facility. The inspection observations were: * failure to reject drug products failing to meet established specifications; * failure to thoroughly review any unexplained discrepancy, the failure of a batch
Biologics
AdvaMed says it supports FDA’s long-standing bioresearch monitoring (BIMO) program and the provision of the FDA Omnibus Reform Act of 2022 that codified the program into law. In its comment on an FDA draft guidance on BIMO inspection processes and practices, AdvaMed says the agency should clarify that
Drugs
FDA has published a guidance, Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases, to help sponsors identify an optimized dosage during clinical development and before applying for approval of a new indication and usage. The agency says the document should be considered
Drugs
FDA has approved an Amneal Pharmaceuticals NDA for Crexont (carbidopa and levodopa) extended-release capsules for treating Parkinson’s disease. The product is described as a novel oral formulation of carbidopa/levodopa that combines both immediate-release granules and extended-release pellets that are designed to provide a longer duration
Drugs
FDA has given accelerated approval to Novartis’ Fabhalta (iptacopan) for reducing proteinuria in primary IgA nephropathy. A company statement says Fabhalta is a first-in-class complement inhibitor for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy at risk of rapid disease progression. The accelerated approval was
Drugs
FDA has cleared a Huxley Medical 510(k) for its at-home sleep apnea diagnostic patch, Sansa. “Unlike current patches, rings, watches, or finger probes, Sansa does not require additional attachments, wires, belts, or hoses,” the company says. “Its patented combination of sensors and materials precisely measures eight physiological channels
Drugs
FDA has published a draft guidance, BCG-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drug and Biological Products for Treatment, with recommendations for developing drug and biological products to treat patients with bacillus Calmette-Guérin (BCG)-unresponsive nonmuscle invasive bladder cancer. The document is intended for drug sponsors, the academic community,
Drugs
FDA has approved a Citius Pharmaceuticals BLA for Lymphir (denileukin diftitox-cxdl) for treating relapsed or refractory (r/r) cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy. The therapy is designed to target the interleukin-2 (IL-2) receptor found on malignant T-cells and Tregs,
Drugs
FDA has approved a Purdue Pharma NDA for Zurnai, a nalmefene hydrochloride auto-injector for the emergency treatment of known or suspected opioid overdose in patients 12 years of age and older. Approval is based on safety and pharmacokinetic studies, and included a study in healthy individuals who use opioids
Drugs
FDA has approved a Servier Pharmaceuticals NDA for Voranigo (vorasidenib), an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor for treating adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma (brain tumors) with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy,
Drugs
CDER Office of Manufacturing Quality compliance officer Yasamain Ameri says data integrity is the biggest issue that is identified by FDA investigators conducting inspections around the world. As reported in the Regulatory Affairs Professionals Society Regulatory Focus online newsletter, Ameri spoke recently at the Orange County (CA) Regulatory Affairs Discussion
Drugs
BeiGene says FDA has “deferred approval” for Tevimbra (tislelizumab) in first-line unresectable, recurrent, locally advanced, or metastatic esophageal squamous cell carcinoma (ESCC) because of a delay in scheduling clinical site inspections. The submission had a July user fee review action date that was missed, and there is no time
Drugs
Novo Nordisk says it has withdrawn a supplemental NDA for its weight-loss drug Wegovy (semaglutide) that is seeking a heart failure indication. “Based on interactions with the FDA, Novo Nordisk has decided to withdraw the file and expects to resubmit the file in the beginning of 2025 with additional