FDA Policy/General
David McFarland
Biologics
Scholar Rock’s Spinal Muscular Atrophy Therapy Approved
FDA Policy/General
FDA Needs Workforce Rebuilding, Less Political Interference: Sinha
Drugs
Etcamah Misses Endpoint in First-Line Breast Cancer Trial
Drugs
FDA Oncology Chief to Focus on AI, Streamlined Drug Development
Oncology Center of Excellence (OCE) director Angelo de Claro says the center will build on initiatives established under longtime and former OCE chief Richard Pazdur while using artificial intelligence (AI) and other approaches to streamline cancer-drug development and increase U.S. participation in clinical trials. Pazdur spent decades overseeing
Drugs
Group Calls for Policy Changes to Sustain Generic, Biosimilar Competition
FDA Policy/General
FDA-SEC Info Sharing Raises Disclosure, Insider Trading Concerns
Drugs
Exelixis NDA Review Extended for Colorectal Cancer Drug
Drugs
Pharming Group’s Joenja Gets Expanded Approval
FDA has granted Pharming Group expanded approval for Joenja (leniolisib) tablets for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in children 4-11 years who weigh at least 27 kg. Joenja was first approved in 2023 for the same indication in patients 12 years and older at a dose
Drugs
Reduced Application Fees Not Sufficient Incentive: Column
FDA’s PDUFA 8 proposal to cut application fees by 50% for qualified drugs to conduct Phase I development in the U.S. is unlikely to achieve that intended result. That’s the opinion of Ovid Therapeutics executive chairman Jeremy Levin. Writing in a BioSpace opinion post, Levin says that
Drugs
Bill to Update FDA Drug Preclusion Rules
Rep. Diana Harshbarger (R-TN) has introduced the Dietary Supplement Innovation Act that she says would modernize FDA’s drug preclusion provision to establish regulatory certainty, ensure pharmaceutical innovation, and support consumer access to dietary supplements. In a statement (includes a link to the bill text and to a brief
Drugs
FDA Open to Alternative Approaches For Psychedelic Drug Trials
FDA says it welcomes novel approaches for evaluating psychedelic drugs for mental health conditions, including changes to how the products are studied, administered and monitored over the long term, agency officials said in a New England Journal of Medicine article. The agency's willingness to consider alternative approaches reflects
enforcement
3 Observations on XeCare FDA-483
FDA has released the Form FDA-483 issued following a 6/8-6/17 inspection at XeCare, a New Albany, OH, producer of sterile and non-sterile drug products. The three inspection observations were: * producing hazardous drugs without providing adequate containment, segregation, and/or cleaning of work surfaces, utensils, and/
Drugs
FDA Partial Clinical Hold on Biohaven Epilepsy Drug
FDA has issued a partial clinical hold on Biohaven’s opakalim (BHV-7000) due to “nonclinical characterization of certain metabolites” documented in rodent testing, according to the company. “It was determined that there was insufficient information related to a specific metabolite to assess risks of that metabolite to human subjects
Devices
BMC Medical Recalls Sleep Apnea Machines
BMC Medical has recalled (Class 1) its Luna G3 APAP (Model LG3600), an auto-adjusting positive airway pressure machine for treating obstructive sleep apnea, due to a firmware defect. If affected devices operate simultaneously under high pressure, high respiratory rate, and high peak flow, an "Error" message on
Drugs
Priority Review for Roche Enspryng sBLA
FDA has granted priority review for a Roche sBLA for Enspryng (satralizumab) to treat myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), an autoimmune disease with no approved treatments. A company statement says FDA is expected to make a decision on the sBLA by 1/10/2027. Roche says acceptance of
enforcement
Hogan Lovells Hires Former FDA Chief Counsel Raza
Former FDA chief counsel Mark Raza has joined Hogan Lovells Cadwalader as senior counsel in its Pharmaceuticals and Biotechnology practice. Raza has more than 35 years of FDA experience, including service in the agency’s Office of Chief Counsel. He retired in 2025 after serving four years as chief counsel.
Drugs
AstraZeneca Reports Positive Data on COPD Biologic
AstraZeneca says data from the Phase 3 OBERON and TITANIA trials showed that tozorakimab is the first biologic to demonstrate statistically significant efficacy in reducing moderate and severe exacerbations in a broad population of patients with chronic obstructive pulmonary disease (COPD) still experiencing exacerbations while on inhaled standard of care.
Drugs
Accelerated Rare Disease Cures Program Strategic Plan Out
FDA has published the Accelerated Rare disease Cures (ARC) program strategic plan covering 2025-2030. The document outlines the vision, mission, and programmatic goals for the five years. “The plan details the specific outcomes, expected results, and related strategies on which ARC’s leadership and the rare disease community will
Drugs
Breakthrough Therapy Designation for UCB’s Cimzia
FDA has granted breakthrough therapy designation to UCB’s Cimzia (certolizumab pegol), a tumor necrosis factor blocker, to prevent placenta-mediated adverse pregnancy outcomes in pregnant women with antiphospholipid syndrome (APS) who are positive for lupus anticoagulant (LA). A company statement says the designation builds on a recent FDA orphan
Biologics
Nanoscope BLA Accepted for Vision Restoration Gene Therapy
FDA has accepted for review a Nanoscope Therapeutics BLA seeking approval for Mogenry (sonpiretigene isteparvovec, MCO-010), an optogenetic gene therapy designed for vision restoration in patients with retinitis pigmentosa (RP) having severe vision loss. “The strength of our RESTORE data, together with the durability we have observed through long-
Drugs
FDA Still Reviewing Rezolute Phase 3 Info
Rezolute says FDA officials have informed it that the agency is continuing to review data from the company’s Phase 3 sunRIZE study of ersodetug to treat hypoglycemia due to congenital hyperinsulinism (HI). A company update statement says that at a March Type B meeting, agency officials asked Rezolute to