Sanofi Rilzabrutinib Wins Japan Orphan Status
Paris, March 2, 2026 Sanofi announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug...
rare disease
QTORIN Rapamycin Meets Phase 3 Endpoint in MLM
WAYNE, Pa., Feb. 24, 2026 — Palvella Therapeutics announced positive topline results from its Phase 3 SELVA clinical study...
FDA CRL Delays Bitopertin Approval for Rare EPP Disease
WATERTOWN, Mass., Feb. 13, 2026 — Disc Medicine announced that the U.S. Food and Drug Administration has issued a...
Ifetroban Gains FDA Fast Track for DMD Heart Disease
NASHVILLE, Tenn., Feb. 4, 2026 —Cumberland Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has granted Fast...
Ultragenyx Gene Therapy Shows Durable Gains in MPS IIIA
NOVATO, Calif., February 3, 2026 — Ultragenyx Pharmaceutical Inc. has reported positive long-term clinical data for UX111, an investigational...
Idorsia Presents Long-Term Lucerastat Data in Fabry Disease
Allschwil, Switzerland – January 30, 2026 — Idorsia Ltd. announced it will present long-term lucerastat clinical data and kidney...
Ipsen Halts Phase II FALKON Trial in Ultra-Rare FOP
Paris, France, December 19, 2025 — Ipsen, a global biopharmaceutical company, announced an update on its pivotal Phase II...
Praxis Data Shows Breakthrough Seizure Control Across Epilepsies
BOSTON, MA — December 8, 2025 — Praxis Precision Medicines presented compelling new clinical results at the American Epilepsy...
Tiakis Advances Tiprelestat into Phase II for Pulmonary Hypertension
Kiel, Germany, December 2, 2025 — Tiakis Biotech has announced that it has successfully completed the required preclinical data...
Tinlarebant Phase 3 Trial Marks Major Breakthrough for STGD1
San Diego, December 1, 2025 — Belite Bio has announced positive topline results from the pivotal global Phase 3...