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...
Rare / orphan disease drugs
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:...
CStone Pharmaceuticals and Blueprint Medicines’ Ayvakit Wins FDA Approval for ISM Treatment
Partners CStone Pharmaceuticals (HKG: 2616) and Blueprint Medicines have announced that the US FDA has...
CANbridge Pharmaceuticals’ Maralixibat Approved by NMPA for Alagille Syndrome
China-based rare disease specialist CANbridge Pharmaceuticals Inc., (HKG: 1228) has announced receiving market approval from...
Coherus and Junshi Biosciences Await FDA Decision on Toripalimab After Inspection
US-based Coherus Biosciences Inc., (NASDAQ: CHRS) has disclosed that the US FDA has recently completed...
Antengene’s ATG-022 Earns FDA Orphan Drug Designations for Gastric and Pancreatic Cancers
China-based Antengene Corporation Limited (HKG: 6996) has announced that its in-house discovered and developed antibody...
Zhejiang Conba Pharmaceutical’s Inhalable Pirfenidone for IPF Clears for Chinese Clinical Trials
Zhejiang Conba Pharmaceutical Co., Ltd (SHA: 600572) has announced that its modified version of US...