Rare Diseases News
🇺🇸 FDAFDA breaking news and regulatory updates for BD, investors, and analysts
Showing 1–12 of 54 articles
Global Regulatory Roundup: Key Updates for June 1, 2026
Fierce Biotech Week: Key Takeaways and Emerging Trends
Palvella Therapeutics Begins Phase 2 Trial of QTORIN Rapamycin for Rare Angiokeratomas
Opus Genetics' OPGx-LCA5 Gene Therapy Accepted Into FDA's Rare Disease Evidence Program
Takeda's TAK-881 Shows Positive Phase 2/3 Results for Primary Immunodeficiency Disease Treatment
Clene's CNM-Au8 Gets FDA Green Light for Accelerated Approval Pathway in ALS Treatment
Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen in McCune-Albright Syndrome
Novartis Receives Health Canada Approval for Fabhalta Oral Treatment for C3G Patients
Mirum Pharmaceuticals VISTAS Study Results for Volixibat in Primary Sclerosing Cholangitis Due May 4, 2026
NIKTIMVO (Axatilimab) Approved in Australia as First-in-Class Chronic Graft-Versus-Host Disease Treatment
Neurocrine Biosciences CRENESSITY Shows Sustained Benefits in Two-Year Pediatric CAH Study
ARCHIMED Acquires Esperion Therapeutics in Strategic Cardiometabolic Deal