United States Food and Drug Administration
FDA Approves Darzalex Faspro for High-Risk Smoldering Multiple Myeloma
FDA approval; Darzalex Faspro; high-risk smoldering multiple myeloma; AQUILA study; early intervention; progression-free survival; daratumumab; hyaluronidase-fihj
Eisai Expands Leqembi Subcutaneous Label, Accelerates Early U.S. Launch Progress for Alzheimer’s Disease
Eisai; Leqembi; subcutaneous autoinjector; label expansion; Supplemental Biologics License Application (sBLA); FDA; maintenance dosing; starting dose; early Alzheimer’s disease; at-home injection; launch progress
Vera Therapeutics Announces Full Phase 3 Win for Atacicept in Kidney Disease, Prepares FDA Filing by Year’s End
Vera Therapeutics; atacicept; Phase 3 trial; IgA nephropathy; proteinuria reduction; UPCR; FDA filing; BLA submission; ASN Kidney Week 2025; autoimmune kidney disease
FDA Rejects Biohaven’s Ataxia Drug Vyglxia, Forcing Company to Cut Costs After Second Approval Failure
Biohaven; FDA rejection; Vyglxia; troriluzole; spinocerebellar ataxia; CRL (Complete Response Letter); clinical trial design; external controls; drug approval; R&D cuts
FDA Unveils Long-Awaited Biosimilar Guidance to Accelerate Approvals and Cut Costs
FDA; biosimilar guidance; 2025 draft guidance; comparative efficacy study; analytical assessment; four-letter suffix; naming convention; biosimilarity; drug costs; biologic medicines
Sarepta’s Duchenne Muscular Dystrophy Drugs Fail Confirmatory Trial, Company Pursues Full FDA Approval Regardless
Sarepta Therapeutics; Duchenne muscular dystrophy; confirmatory trial; AMONDYS 45; VYONDYS 53; Elevidys; FDA approval; clinical trial failure; accelerated approval; statistical significance
UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease: Kygevvi for Thymidine Kinase 2 Deficiency (TK2d)
UCB; FDA approval; Kygevvi; mitochondrial disease; Thymidine Kinase 2 deficiency; TK2d; doxecitine; doxribtimine; rare diseases; Orphan Drug; Breakthrough Therapy; Priority Review; Rare Pediatric Disease
Recent News: Live at 11 a.m. ET – M&A, FDA, MFN and Collaboration with FT
M&A; FDA; MFN; Financial Times; mergers and acquisitions; drug approval; economics; live news
uniQure Faces Delay as FDA Walks Back Accelerated Approval for Huntington’s Gene Therapy
uniQure; AMT-130; Huntington’s disease; gene therapy; FDA; accelerated approval; regulatory setback; clinical trial
‘Trying to take me out’: Tidmarsh’s exit at FDA pushes an agency in transition toward turmoil
FDA; George Tidmarsh; resignation; personal conduct probe; Kevin Tang; Center for Drug Evaluation and Research (CDER); agency turmoil; Vinay Prasad; leadership transitions; regulatory authority