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...
Rare / orphan disease drugs
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,...
Roche Imposes New Dosing Restrictions on Elevidys for Non-Ambulatory DMD Patients
Swiss pharmaceutical giant Roche AG (SWX: ROG, OTCMKTS: RHHBY) announced immediate new dosing restrictions for...
Sanofi’s Rilzabrutinib Shows Promise in IgG4-Related Disease Trial
French pharmaceutical giant Sanofi (NASDAQ: SNY) announced positive results from a Phase II study of...
Biogen Launches Qalsody in China for SOD1-ALS Treatment
US-based Biogen (NASDAQ: BIIB) officially launched Qalsody (tofersen) in China this week. The drug, the...
Sanofi’s Rilzabrutinib Granted Orphan Drug Designation for Sickle Cell Disease
France-based Sanofi (NASDAQ: SNY) announced that it has received an additional Orphan Drug Designation (ODD)...
Sanofi to Acquire Blueprint Medicines for $9.1 Billion to Expand Rare Disease Pipeline
French pharmaceutical giant Sanofi (NASDAQ: SNY) announced plans to acquire US-based Blueprint Medicines Corporation for...
R&B Biotech Receives FDA Approval for ZS805 Gene Therapy in Fabry Disease
Sichuan R&B Biotech, a China-based biotech firm, announced receiving clinical approval from the US Food...
Novartis’ Fabhalta Approved for Previously Treated PNH Patients in China
The China unit of Swiss pharmaceutical giant Novartis (NYSE: NVS) announced that the National Medical...