Belief BioMed Gains NMPA Clinical Trial Approval for DMD Gene Therapy BBM-D101
China-based gene therapy specialist Belief BioMed Inc. (BBM) has announced that it has received clinical...
Rare / orphan disease drugs
Biogen’s High-Dose Spinraza Regimen Accepted for CDE Review in China
US-based Biogen Inc. (NASDAQ: BIIB) announced that the Center for Drug Evaluation (CDE) in China...
Takeda’s Adzynma, BeiGene’s Sonrotoclax, and Avistone’s Andatinib Set for Priority Review in China
The website of China’s Center for Drug Evaluation (CDE) indicates that Takeda’s (TYO: 4502) Adzynma...
Belief BioMed and Takeda Win NMPA Approval for China’s First Hemophilia B Gene Therapy
China-based gene therapy specialist Belief BioMed Inc. and Takeda’s (NYSE: TAK, TYO: 4502) China unit...
HemaCell’s XJ-MK-002 Gains FDA Orphan Drug Designation for Giant Platelet Syndrome
China-based Suzhou Xueji Biotechnology Co., Ltd (HemaCell), a pioneer in platelet regeneration-focused cell therapy, announced...
Sanofi’s Rilzabrutinib Granted Orphan Drug Designation for Rare Diseases
Sanofi (EPA: SAN, NASDAQ: SNY) announced that it has received Orphan Drug Designation (ODD) from...
Sarepta and Roche Seek to Resume Elevidys Trials After EMA Halt
The European Medicines Agency (EMA) may lift the temporary hold on certain Phase III studies...
Hengrui Pharmaceuticals Secures Clinical Trial Approval for HRS-9813 in IPF
Hengrui Pharmaceuticals (SHA: 600276) announced on April 1, 2025, that its HRS-9813 capsule has received...
Sanofi’s Qfitlia Approved by FDA for Hemophilia A and B Prophylaxis
France-based Sanofi (EPA: SAN, NASDAQ: SNY) has announced receiving marketing approval from the US Food...
Luye Pharma’s BA1302 Receives FDA Orphan Drug Designation for Lung and Pancreatic Cancer
China-based Luye Pharma Group’s (HKG: 2186) controlling subsidiary Shandong Boan Biotechnology Co., Ltd (HKG: 6955)...
Sanofi’s Tolebrutinib Accepted for FDA Review in Non-Relapsing Secondary Progressive Multiple Sclerosis
Sanofi (EPA: SAN, NASDAQ: SNY) announced that a market filing for its tolebrutinib as a...
SineuGene Therapeutics’ SNUG01 Receives FDA Clearance for ALS Clinical Study
Beijing-based SineuGene Therapeutics, a developer of gene therapies for brain disorders, announced last week that...
Suzhou Xueji Biotech’s XJ-MK-002 Gets FDA Orphan Drug Designation for CAMT
China-based cell therapy developer Suzhou Xueji Biotechnology Co., Ltd’s (HemaCell) XJ-MK-002, described as the world’s...
Novartis’ Fabhalta Approved by FDA for C3 Glomerulopathy Treatment
Swiss pharmaceutical giant Novartis’ (NYSE: NVS) iptacopan, marketed as Fabhalta, has received approval from the...
AccurEdit Therapeutics Receives FDA Orphan Drug Designation for ATTR Treatment
China-based AccurEdit Therapeutics (Suzhou) Co., Ltd announced receiving Orphan Drug Designation (ODD) from the US...
Johnson & Johnson’s Nipocalimab Receives FDA Fast Track Designation for Sjögren’s Disease
US giant Johnson & Johnson (J&J; NYSE: JNJ) announced receiving Fast Track designation (FTD) from...
Merck & Co.’s Winrevair Approved for Pulmonary Arterial Hypertension in Hainan
US-based giant Merck & Co., Inc. (NYSE: MRK) announced receiving approval from the Hainan Medical...
Visirna Therapeutics Announces Positive Phase III Results for Plozasiran in FCS Treatment
Shanghai-based Visirna Therapeutics announced positive topline results from the Phase III study of plozasiran in...
Lundbeck’s Amlenetug Receives Orphan Drug Designation in Japan for Multiple System Atrophy
Denmark-based pharmaceutical company Lundbeck A/S (OTCMKTS: HLUBF) announced receiving Orphan Drug Designation (ODD) from Japan’s...
Suzhou GenAssist’s GEN6050X Receives FDA Clearance for Duchenne Muscular Dystrophy Treatment
China-based Suzhou GenAssist Therapeutic Co., Ltd has announced receiving clearance from the US Food and...