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...
Rare / orphan disease drugs
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...
CDE Launches SPARK Project to Boost Pediatric Antitumor Drug R&D
China’s Center for Drug Evaluation (CDE) has released a notification to solicit feedback on its...
Huadong Medicine’s ADC HDM2005 Granted FDA Orphan Drug Designation for MCL
China-based Huadong Medicine Co., Ltd (SHE: 000963) announced that its antibody-drug conjugate (ADC) HDM2005 has...
InnoCare Pharma’s Orelabrutinib Shows Efficacy in RRMS at ACTRIMS Forum
Beijing-based InnoCare Pharma (HKG: 9969, SHA: 688428) announced the presentation of Phase II study results...
YolTech Therapeutics’ YOLT-203 Shows Promise in Primary Hyperoxaluria Type 1 Trial
YolTech Therapeutics, a Shanghai-based startup specializing in lipid nanoparticle delivery and gene editing, announced positive...
CANbridge Pharmaceuticals Doses First Patient in CAN108 Biliary Atresia Study
China-based rare disease specialist CANbridge Pharmaceuticals Inc. (HKG: 1228) announced that the first patient has...
CDE Includes Chiral Tech’s Cerdelga Generic in Rare Disease Care Plan
China’s Center for Drug Evaluation (CDE) announced the inclusion of Beijing Chiral Tech’s generic version...
Pfizer’s Hympavzi (Marstacimab) Launched in China’s Boao Lecheng Pilot Zone for Hemophilia
Pfizer Inc. (NYSE: PFE) announced the implementation of its hemophilia therapy Hympavzi (marstacimab) in China’s...
Takeda’s Takhzyro Receives EMA Approval for New Administration Form in HAE Patients
Japan-headquartered Takeda (NYSE: TAK, TYO: 4502) announced that the European Medicines Agency (EMA) has approved...
Innorna’s IN013 Receives FDA Rare Pediatric Designation for Wilson’s Disease Treatment
Shenzhen-based Innorna Co., Ltd, a platform company leveraging mRNA and lipid nanoparticle (LNP) delivery technologies,...
Gene Cradle and Byongen Therapeutics Partner on Next-Generation DMD Gene Therapies
Gene Cradle and Byongen Therapeutics, both Beijing-based gene therapy specialists, have formed a strategic alliance...