SSY Group Ltd Receives NMPA Approval for Stiripentol Dry Suspension for Dravet Syndrome
China-based SSY Group Ltd (HKG: 2005) has announced that it has received marketing approval from...
Rare / orphan disease drugs
Novo Nordisk’s Turoctocog Alfa Pegol for Hemophilia A Accepted for Review by China’s CDE
The Center for Drug Evaluation (CDE) in China has indicated on its website that the...
AstraZeneca’s Eplontersen Gains Tacit Approval from CDE for ATTR-CM Clinical Study
The Center for Drug Evaluation (CDE) website indicates that UK pharmaceutical major AstraZeneca’s (AZ, NASDAQ:...
BGI Genomics Partners with Thai Entities to Combat Thalassemia through Genomics and Cell Therapy
China-based genomics leader BGI Genomics Co., Ltd (SHE: 300676) has entered into a memorandum of...
Zai Lab’s Vyvgart Receives NMPA Review for Subcutaneous Injection Form in gMG Treatment
China-based Zai Lab Ltd (NASDAQ: ZLAB; HKG: 9688) has announced that the National Medical Products...
NMPA Approves Pitolisant for Narcolepsy-Related Excessive Daytime Drowsiness and Cataplexy
The National Medical Products Administration (NMPA) has approved pitolisant as a treatment for excessive daytime...
RareStone Group’s Pitolisant Earns Priority Review Status for Narcolepsy Treatment in China
The Center for Drug Evaluation (CDE) website in China has indicated that RareStone Group, a...
Shanghai Pharmaceuticals’ MediTrust Health Partners with Yuanda Shuyang on Rare Disease Treatment Model
Shanghai Pharmaceuticals Holding Co., Ltd’s (SHA: 601607, HKG: 2607) subsidiary, MediTrust Health, which specializes in...
Beijing Tiantan Biological Completes Phase I Study for Recombinant Coagulation Factor VIIa
China-based Beijing Tiantan Biological Products Co., Ltd (SHA: 600161) has announced the conclusion of a...
CANbridge Pharmaceuticals Reports Positive Phase Ib Results for Omoprubart in PNH Treatment
China-based rare disease firm CANbridge Pharmaceuticals Inc., (HKG: 1228) has published the preliminary results of...
Sarepta Therapeutics’ Elevidys Receives FDA Accelerated Approval for Duchenne Muscular Dystrophy
US-based Sarepta Therapeutics, Inc. (NASDAQ: SRPT) has announced that the U.S. Food and Drug Administration...
WinHealth Pharma Receives NMPA Approval for Glycerol Phenylbutyrate for Urea Cycle Disorders
China-based Hong Kong WinHealth Pharma Group Ltd has announced receiving market approval from the National...
Roche’s Evrysdi Receives NMPA Approval for Wider SMA Patient Age Range
Swiss pharmaceutical giant Roche (SWX: ROG) has announced that its blockbuster nervous system drug Evrysdi...
Sperogenix Therapeutics and LUCA Healthcare Join Forces to Advance Digital Therapy for Myasthenia Gravis
China-based rare disease specialist Sperogenix Therapeutics Limited has entered into a partnership with compatriot firm...
China Medical System’s Diazepam Nasal Spray Receives NMPA Approval for Cluster Seizures
China-based China Medical System Holdings Ltd (CMS; HKG: 0867) has announced receiving New Drug Application...
AstraZeneca’s Soliris Secures Third Indication for Myasthenia Gravis in China
UK pharmaceutical giant AstraZeneca (AZ, NASDAQ: AZN) has announced that its Soliris (eculizumab), a first-in-class...
Hainan Boao Lecheng Partners with Recordati on Rare Disease Drug Development
The Hainan Boao Lecheng International Medical Tourism Pilot Zone has announced a strategic partnership with...
BioMarin Pharmaceutical to Discontinue Vimizim Import License in China
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced its decision not to renew the Import Drug...
Biosyngen’s BRG01 Earns Orphan Drug Designation from US FDA for Nasopharyngeal Carcinoma
Biosyngen, a leading cell therapy developer based in Guangzhou, has announced that it has received...
Novartis’s Iptacopan on Track for Priority Review Status in China for PNH Treatment
China’s Center for Drug Evaluation (CDE) website has indicated that Swiss pharmaceutical giant Novartis’ (NYSE:...