United States Food and Drug Administration
Dyne Therapeutics Reports Positive Results for Duchenne Drug DYNE-251, Plans FDA Approval Submission
Dyne Therapeutics; DYNE-251; Duchenne muscular dystrophy; exon 51 skipping; DELIVER trial; FDA Breakthrough Therapy Designation; Biologics License Application; accelerated approval
FDA Signals Higher Efficacy Bar for New CAR‑T Cancer Therapies
FDA; CAR-T; chimeric antigen receptor T-cell therapy; oncology; regulatory standards; superiority trials; randomized controlled trials; overall survival; hematologic malignancies; cell and gene therapy
Rapport Therapeutics Reports Updated Phase 2a Data for Seizure Drug RAP-219
Rapport Therapeutics; RAP-219; Phase 2a trial; seizure drug; epilepsy; focal onset seizures; clinical seizure reduction; FDA meeting; Phase 3 trials
UniQure restates FDA’s negative feedback on Huntington’s trial; Neurimmune expands AstraZeneca alliance
UniQure; AMT-130; FDA feedback; external control group; gene therapy; Neurimmune; AstraZeneca; neurodegeneration; antibody collaboration; licensing deal; biopharmaceutical partnerships
FDA names Tracy Beth Heg as acting CDER chief
Tracy Beth Heg; acting CDER chief; FDA leadership; Center for Drug Evaluation and Research; COVID-19 vaccine safety; drug regulation; U.S. Food and Drug Administration
Former FDA Chiefs Criticize Proposed Vaccine Policy Changes at ACIP Meeting
former FDA commissioners; CDC; ACIP meeting; vaccine policy changes; COVID-19 vaccines; Vinay Prasad; Center for Biologics Evaluation and Research; vaccine safety regulation; Senator Bill Cassidy; New England Journal of Medicine commentary
FDA Refuses to Release Detailed Data on Claimed COVID Vaccine-Linked Child Deaths in Near Term
FDA; COVID-19 vaccine; child deaths; myocarditis; vaccine safety; Vinay Prasad; data release; vaccine oversight
Capricor Therapeutics Reports Positive Phase 3 HOPE-3 Trial Results for Deramiocel in Duchenne Muscular Dystrophy, Planning FDA Resubmission
Capricor Therapeutics; Deramiocel; Duchenne muscular dystrophy; HOPE-3 trial; Phase 3 clinical trial; FDA rejection; cardiac function preservation; upper limb function; clinical trial results; regulatory approval pathway
Recent Developments on Vinay Prasad’s Leaked FDA Memo, Alzheimer’s Disease Updates, and Next-Gen Obesity Drugs
Vinay Prasad FDA memo; COVID-19 vaccine deaths children; FDA vaccine approval changes; Alzheimer’s disease research 2025; obesity drugs next generation; COVID-19 vaccine safety controversy; FDA vaccine policy overhaul
Top Drug Regulator Richard Pazdur to Retire from FDA Amid Industry Concerns
Richard Pazdur; FDA; retirement; Center for Drug Evaluation and Research; drug regulation; Marty Makary; leadership turnover