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...
Orphan Drug
Advertisement
FDA Grants Fast Track to AJ201 for Kennedy’s Disease Therapy
TAIPEI, Taiwan – October 23, 2025 AnnJi Pharmaceutical Co., Ltd. (AJ Pharm) announced that the U.S. Food and Drug...
Oculis Highlights Late-Stage Eye Pipeline at AAO 2025
October 14, 2025 | Zug, Switzerland — Oculis Holding AG (Nasdaq: OCS), a global biopharmaceutical company advancing therapies for...
Star Therapeutics Raises $125M to Advance Bleeding Disorder Therapy
SOUTH SAN FRANCISCO, Calif., Sept. 30, 2025 — Star Therapeutics announced it has closed an oversubscribed $125 million Series...
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)...
Advertisements
Intellia Completes Phase 3 Enrollment for HAE Gene Therapy
September 18, 2025 — Cambridge, Massachusetts Intellia has completed enrollment in the global Phase 3 HAELO trial of lonvoguran...
Health Canada Approves Bylvay for Cholestatic Pruritus in ALGS
OTTAWA, Canada, September 9, 2025 — Health Canada has granted approval to Bylvay™ (odevixibat) for the treatment of cholestatic...
REGENXBIO Reports Positive 12-Month Data from RGX-121 CAMPSIITE® Trial in MPS II
EGENXBIO Inc. (Nasdaq: RGNX) announced positive 12-month pivotal data from its Phase I/II/III CAMPSIITE® trial of RGX-121 (clemidsogene lanparvovec),...
Zydus Therapeutics Reports Positive Topline Results from EPICS-III Trial of Saroglitazar Magnesium in Primary Biliary Cholangitis
Zydus Therapeutics, Inc., a U.S.-based innovation arm of Zydus Lifesciences Ltd., today announced positive topline data from its pivotal...
Advertisement
