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)...
Rare / orphan disease drugs
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...
RemeGen Inks Global Licensing Pact With Vor Biopharma for Telitacicept
China-based RemeGen Co., Ltd. (SHA: 688331, HKG: 9995) announced an exclusive licensing agreement with U.S.-based...
Biogen’s Phase I Results Highlight Salanersen’s Potential in Spinal Muscular Atrophy
Biogen (NASDAQ: BIIB) announced this week topline results from the Phase I study of salanersen...
SineuGene’s ALS Gene Therapy SNUG01 Granted FDA Orphan Drug Designation
Beijing-based SineuGene Therapeutics, a developer of gene therapies for brain disorders, announced this week that...
Genevector’s JWK002 Gains NMPA IND Approval for XLRS Gene Therapy
China-based Chengdu Geneector Biotechnology Co., Ltd. (Genevector), a developer of AAV gene therapy drugs, announced...
Lundbeck’s Lu AG13909 Wins Orphan Drug Designations for Congenital Adrenal Hyperplasia
Denmark-based pharmaceutical company Lundbeck A/S (OTCMKTS: HLUBF) announced that it has received Orphan Drug Designations...
Zai Lab’s Partner argenx Secures EU Approval for Vygart in CIDP Treatment
China-based Zai Lab Limited’s (NASDAQ: ZLAB, HKG: 9688) Dutch partner argenx SE (NASDAQ: ARGX) announced...
RemeGen’s Telitacicept Receives Orphan Drug Designation for Myasthenia Gravis in Europe
China-based RemeGen Co., Ltd. (SHA: 688331, HKG: 9995) announced that it has received orphan drug...
Suzhou GenAssist’s GEN6050X Gains FDA Orphan Drug Status for Duchenne Muscular Dystrophy
China-based Suzhou GenAssist Therapeutic Co., Ltd. announced that its in vivo base editing therapy, GEN6050X,...