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...
Rare / orphan disease drugs
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...
Yifan Pharma’s Generic Glycerol Phenylbutyrate Accepted for NMPA Review
China-based Yifan Pharmaceutical Co., Ltd (SHE: 002019) announced that the National Medical Products Administration (NMPA)...
Hainan Boao Research Hospital Secures Approval for China’s First Medical-Food Import for Rare Metabolic Disorders
Hainan Boao Research Hospital, operated by Shanghai’s Ruijin Hospital, has obtained provincial approval to import...
Ningbo Health Gene and Biofast Biotech Partner to Advance Genetic Testing for Hereditary Diseases
Chinese firms Ningbo Health Gene Technologies Co., Ltd. and Biofast Biotechnology Co., Ltd. announced late...
Golden Age Health Secures Exclusive Rights to Innostellar’s Gene Therapy LX-101 in China
Singapore-based Golden Age Health, a specialty pharmaceutical company focused on rare and specialty diseases, has...
Zelgen’s ZG006 Receives Breakthrough Therapy Designation for Advanced Small Cell Lung Cancer
China-based Suzhou Zelgen Biopharmaceuticals Co., Ltd (SHA: 688266) announced that it has received breakthrough therapy...
IASO Biotherapeutics Receives Orphan Drug Designation in South Korea for Fucaso in Multiple Myeloma
China-based IASO Biotherapeutics announced that its BCMA-targeted chimeric antigen receptor (CAR) T cell therapy, Fucaso...
CStone Pharmaceuticals Announces Phase III Trial Progress for Nofazinlimab in Hepatocellular Carcinoma
China-based CStone Pharmaceuticals (HKG: 2616) announced the latest progress in its global multicenter Phase III...
MSD’s Winrevair Accepted for Priority Review by FDA for Pulmonary Arterial Hypertension
US pharmaceutical giant Merck, Sharp & Dohme (MSD; NYSE: MRK) announced that the US Food...
CSPC Pharma Receives NMPA Approval for Hydroxocobalamine in Pediatric MMA Treatment
China-based CSPC Pharmaceutical Group Ltd (HKG: 1093) announced that it has received clinical trial approval...
CANbridge’s Geruining Approved for Gaucher Disease Treatment: First Domestically Developed ERT
China-based CANbridge Pharmaceuticals Inc., (HKG: 1228) announced that the first prescription of its Geruining (velaglucerase...
UCB’s Fenfluramine Shows Positive Results in Phase III GEMZ Study for CDKL5 Deficiency Disorder
Belgium-based biopharma UCB (EBR: UCB) announced positive results from its Phase III GEMZ study, evaluating...
Sanofi’s Riliprubart Granted Orphan Drug Status for Antibody-Mediated Rejection Treatment
France-based major Sanofi (NASDAQ: SNY) has secured Orphan Drug Designation (ODD) from the US Food...