Infrequent
EMA Identifies Very Rare Eye Condition as Side Effect of Semaglutide
EMA; semaglutide; rare side effect; NAION; vision loss; Ozempic; Wegovy; Rybelsus
Sarepta’s rAAVrh74 Viral Vector Receives FDA Platform Technology Designation for Gene Therapy
Sarepta Therapeutics; FDA; platform technology designation; rAAVrh74 viral vector; gene therapy; SRP-9003; limb-girdle muscular dystrophy; rare diseases
Sanofi to Acquire Blueprint Medicines for Up to $9.5 Billion, Expanding Immunology Focus
Sanofi; Blueprint Medicines; acquisition; immunology; systemic mastocytosis; Ayvakit; elenestinib; rare diseases; biopharma; pipeline
FDA Rejects Stealth BioTherapeutics’ Rare Disease Drug but Leaves Door Open for Accelerated Approval
FDA rejection; Stealth BioTherapeutics; elamipretide; Barth syndrome; rare disease; accelerated approval; muscle strength endpoint; phase 2 trial; regulatory delay
Applied Therapeutics’ Rare Disease Drug Fails Another Phase 3 Trial After FDA Rejection
Applied Therapeutics; govorestat; rare disease; phase 3 failure; FDA rejection; classic galactosemia; aldose reductase inhibitor; clinical trial conduct; FDA warning letter
BioMarin Acquires Inozyme Pharma for $270M, Gaining Phase 3 Rare Disease Therapy
BioMarin; Inozyme Pharma; acquisition; rare disease; INZ-701; ENPP1 Deficiency; enzyme replacement therapy; Phase 3 clinical trial
FDA Delays Decision on Biohaven’s Troriluzole for Rare Disease, Plans Advisory Committee Review
Biohaven; FDA delay; troriluzole; spinocerebellar ataxia (SCA); rare disease drug; PDUFA extension; advisory committee; neurodegenerative disease
J&J’s $130M gene therapy bet flunks rare retinal disease trial
Johnson & Johnson, gene therapy, bota-vec, X-linked retinitis pigmentosa, Phase 3 failure, vision loss treatment, rare eye disease, $130 million investment
FDA chief Makary talks expediting drug approvals, RIFs and the abortion pill
United States Food and Drug Administration, Califf, Unspecified Abortion, Contraceptives, Oral, Accelerated, Artificial Intelligence, RIFs, Infrequent, Agencies
Abeona’s $3.1M gene therapy wins FDA nod for rare skin disease
United States Food and Drug Administration, United States Food and Drug Administration, Infrequent, ZEVASKYN, Abeona, gene therapy, Dermatologic disorders, Hallopeau-Siemens Disease, Approved, Abeona ‘s