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),...
Rare / orphan disease drugs
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...
Humanwell Healthcare Receives NMPA Approval for HW241045 IPF Clinical Trials
China-based Humanwell Healthcare (Group) Co., Ltd. (SHA: 600079) announced that its wholly-owned subsidiary, Humanwell Innovation...
AstraZeneca’s Ultomiris Gains NMPA Approval for Neuromyelitis Optica Spectrum Disorder
UK-based pharmaceutical giant AstraZeneca (AZ; NASDAQ: AZN) announced that China’s National Medical Products Administration (NMPA)...
Sanofi’s SAR446523 Gains FDA Orphan Drug Status for Relapsed or Refractory Multiple Myeloma
French pharmaceutical major Sanofi (NASDAQ: SNY) reported that the U.S. Food and Drug Administration (FDA)...
Zhaoke Ophthalmology’s Melphalan Granted FDA Orphan Drug Designation for Pediatric Retinoblastoma
China-based Zhaoke Ophthalmology Ltd (HKG: 6622), a leader in ophthalmic solutions, today announced that its...
YolTech’s YOLT-203 Gains Orphan Drug Designation for Primary Hyperoxaluria Type 1 Treatment
China-based YolTech Therapeutics announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products...
Alphamab Oncology’s JSKN003 Granted FDA Orphan Drug Designation for Gastric and Gastroesophageal Junction Cancer
China-based Alphamab Oncology (HKG: 9966) announced today that its drug candidate JSKN003 has received Orphan...
Roche’s Elevidys Receives Negative CHMP Opinion for Duchenne Muscular Dystrophy
Swiss giant Roche AG (SWX: ROG, OTCMKTS: RHHBY) announced today that the Committee for Medicinal...
Sarepta Therapeutics Announces 500 Employee Layoffs After FDA Elevidys Trial Suspension
US-based Sarepta Therapeutics, a subsidiary of Sanofi (NASDAQ: SNY), announced the layoff of approximately 500...