FDA Accepts BioMarin’s PALYNZIQ sBLA for Priority Review in Adolescents with PKU
San Rafael, California — October 30, 2025 — BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration...
rare disease
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Halia Therapeutics’ Ofirnoflast Gains FDA Orphan Drug Status for MDS
LEHI, Utah, Oct. 23, 2025 — Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on therapies targeting the root...
Stealth BioTherapeutics Expands Access with Mito Assist™ and AnovoRx Partnership
NEEDHAM, Mass., October 9, 2025 — Stealth BioTherapeutics Inc. has announced the launch of Mito Assist™, a comprehensive patient...
FDA Breakthrough Status Advances Rett Syndrome Gene Therapy
DALLAS, Oct. 2, 2025 — The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to TSHA-102,...
Sobi Taps Kinaxis Platform to Speed Rare Disease Therapy Supply
OTTAWA, Oct. 2, 2025 — Kinaxis announced that Sobi, a specialist in rare‐disease biopharma, will adopt the Kinaxis Maestro®...
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Larimar’s Nomlabofusp Shows Long-Term Promise in Friedreich’s Ataxia
Bala Cynwyd, Pa., September 29, 2025: Larimar Therapeutics announced positive new data from an ongoing long-term open-label study of...
FDA Accelerates First-Ever Barth Syndrome Therapy, FORZINITY™
NEEDHAM, Mass., September 19, 2025 – Stealth BioTherapeutics Inc. today announced that the U.S. Food and Drug Administration (FDA)...
FDA Grants Fast Track Status to NS-229 for Rare Autoimmune Disease
PARAMUS, NJ, September 9, 2025 — The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to...
UK Approves Once-Monthly Octreotide Injection for Acromegaly Care
Lund, Sweden — August 28, 2025 — The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval...
FDA Approves First RNA Therapy for Hereditary Angioedema Prophylaxis
CARLSBAD, Calif., August 21, 2025 — The U.S. Food and Drug Administration (FDA) has approved DAWNZERA™ (donidalorsen), the first...
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