UCB Biosciences Receives NMPA Marketing Authorization for Zilucoplan (Zilbrysq) in Adult Generalized Myasthenia Gravis
UCB S.A. (EBR: UCB), a global biopharmaceutical company, announced today that the National Medical Products...
Rare / orphan disease drugs
ReviR Therapeutics Secures FDA Orphan Drug Designation for RTX‑117, Targeting Charcot‑Marie‑Tooth Disease
ReviR Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan...
Boehringer Ingelheim Secures FDA Approval for Nerandomilast (JASCAYD) in Idiopathic Pulmonary Fibrosis
Boehringer Ingelheim (BI) announced today that the U.S. Food and Drug Administration (FDA) has approved...
Ipsen Launches BYLVAY in China for Rare Liver Disease
France‑based Ipsen S.A. (EPA: IPN, OTCMKTS: IPSEY) announced the official commercial launch of its rare‑disease...
Biogen Receives FDA Complete‑Response Letter on High‑Dose Nusinersen for SMA
Biogen Inc. (NASDAQ: BIIB) announced today that the U.S. Food & Drug Administration (FDA) has...
Stealth BioTherapeutics Receives FDA Accelerated Approval for Forzinity in Barth Syndrome
The U.S. Food and Drug Administration (FDA) granted accelerated approval to Stealth BioTherapeutics’ Forzinity (elamipretide)...
Biogen Acquires Alcyone Therapeutics to Drive ThecaFlex DRx Implant Development
Biogen Inc. (NASDAQ: BIIB) announced the acquisition of Massachusetts‑based Alcyone Therapeutics. The deal centers on...
Baheal’s 24% Take‑over of Tianjin Jikun Secures Rights to Pulmonary Fibrosis Drug JK1033
China‑based Baheal Pharmaceutical Group (SHE: 301015) announced a strategic cooperation agreement with Tianjin Jikun Pharmaceutical...
FDA Unveils RDEP to Accelerate Rare Disease Therapy Development
The U.S. Food and Drug Administration (FDA) today issued the Rare Disease Evidence Principles (RDEP),...
Enlaza‑Vertex Partner on $45 M Multi‑Target Drug Discovery Collaboration
Enlaza Therapeutics, Inc. and Vertex Pharmaceuticals, Inc. (NASDAQ: VRTX) today announced a multi‑target drug discovery...
Bioheng Therapeutics Receives FDA RPDD for Off‑The‑Shelf CAR‑T CTD402
Bioheng Therapeutics (China) announced that the U.S. Food and Drug Administration (FDA) has granted Rare...
Changchun High Files GenSci134 Clinical Trial for Growth Hormone Deficiency
Changchun High & New Technology Industries (Group) Inc. (SHE: 000661) on Aug 27, 2025 disclosed...
Regeneron’s Cemdisiran Beats Key Milestones in Phase III gMG Trial
US-based Regeneron Pharmaceuticals Inc. (NASDAQ: REGN) on Aug 27, 2025 disclosed that its investigational monotherapy, cemdisiran,...
Ionis Pharmaceuticals’ DAWNZERA Approved by FDA for HAE Prevention
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced on August 22, 2025, that the U.S. Food and...
Santhera Pharmaceuticals Signs Exclusive Agreement with Uniphar for AGAMREE Distribution in Middle East
Swiss firm Santhera Pharmaceuticals (SWX: SANN), a leader in innovative therapies for rare diseases, has...
Sanofi’s Rilzabrutinib Granted Orphan Drug Designation for IgG4-Related Disease by EMA
French pharmaceutical major Sanofi (NASDAQ: SNY) announced that it has received another orphan drug designation...
CANbridge Pharmaceuticals Secures HK 100 Million Equity Financing From Baheal Pharma
CANbridge Pharmaceuticals (HKG: 1228) announced on August 13, 2025, that it has entered into a...
Hengrui Pharmaceuticals Expands Hetrombopag Olamine’s Indication for ITP Patients
China-based Jiangsu Hengrui Pharmaceuticals Co., Ltd. (HKG: 1276, SHA: 600276) announced that the indication expansion for...
Novartis Evaluates Acquisition of Avidity Biosciences to Strengthen Rare Disease Pipeline
According to reports from several news organizations citing insider sources, that Novartis AG (NYSE: NVS)...
Hengrui’s Trastuzumab Rezetecan Granted FDA Orphan Drug Designation for Gastric Cancer Treatment
China-based Jiangsu Hengrui Pharmaceuticals Co., Ltd. (HKG: 1276, SHA: 600276) announced that it has received...