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...
Rare / orphan disease drugs
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...
Biogen Licenses Stoke Therapeutics’ Zorevunersen for Dravet Syndrome Outside Americas
US-based Biogen Inc. (NASDAQ: BIIB) has entered into a licensing agreement with Stoke Therapeutics, Inc....
Merck’s Welireg Wins Conditional EU Approval for VHL Disease and Advanced RCC
US pharmaceutical giant Merck, Sharp & Dohme (MSD, NYSE: MRK) announced that it has received...
Wanbangde New Building Materials’ Mecobalamin Earns FDA Orphan Drug Designation for ALS
China’s Wanbangde New Building Materials Co., Ltd (SHE: 002082) announced that its mecobalamin product has...
Bayer’s Acoramidis (Beyonttra) Gets EMA Marketing Approval for ATTR-CM Treatment in EU
Germany-headquartered Bayer (ETR: BAYN) has received marketing approval from the European Medicines Agency (EMA) for...
Roche Transfers InterMune and Esbriet to Legacy Pharma in IP Deal
Swiss pharma giant Roche (SWX: ROG) has transferred US-based InterMune, Inc., acquired for USD 8...
Sinopep-Allsino Wins NMPA Approval for Generic Liptruzet in China
Jiangsu-based CDMO Sinopep-Allsino Biopharmaceutical Co., Ltd (SHA: 688076) has received marketing approval from China’s National...
Regend Therapeutics’ Pulmovinci Receives FDA Orphan Drug Designation for IPF
Suzhou-based stem cell therapy specialist Regend Therapeutics announced that the US Food and Drug Administration...
Novo Nordisk’s Mim8 Shows Promise in Hemophilia A Trial with 98% Caregiver Preference
Denmark-based Novo Nordisk A/S (NYSE: NVO) released interim results from the Phase III FRONTIER3 study,...
Betta Pharmaceuticals Gains NMPA Approval for BPI-452080 Clinical Study
China-based Betta Pharmaceuticals Co., Ltd (SHE: 300558) announced that it has received approval from the...
Sinopharm Partners with Biogen for Comprehensive Service Agreement
China-based Sinopharm Group Co., Ltd (HKG: 1099) has entered into a strategic partnership with US-based...