Humanwell Healthcare Initiates Phase IIa Clinical Study for IPF Drug HW021199
China-based Humanwell Healthcare (Group) Co., Ltd (SHA: 600079) has announced the commencement of a Phase...
Rare / orphan disease drugs
GC Biopharma Presents Data Showing Higher Thrombotic Risk with Roche’s Hemlibra
South Korea’s GC Biopharma (KRX: 006280) has presented evidence suggesting that Roche’s (SWX: ROG) hemophilia...
Immunofoco’s IMC008 Earns Orphan Drug Designation for Gastric Cancer from FDA
Suzhou-based cell therapy developer Immunofoco has announced that it has received orphan drug designation (ODD)...
Novartis Acquires Chinook Therapeutics for $3.2 Billion, Eyes Kidney Disease Market
Switzerland-based pharmaceutical major Novartis (NYSE: NVS) has successfully completed the scheduled purchase of US-based kidney...
Beijing’s Gene Cradle Secures Pre-Series B+ Funding Led by Cash Capital
Gene Cradle, a Beijing-based gene therapy specialist, has reportedly secured tens of millions of renminbi...
ABM Therapeutics’ ABM-1310 Earns Orphan Drug Designation for BRAF V600 Mutant Glioblastoma
Shanghai-based ABM Therapeutics Inc. has announced that it has received Orphan Drug Designation (ODD) from...
CDE Issues Guiding Principles for Natural History Research in Rare Disease Drug Development
The Center for Drug Evaluation (CDE) has released the “Guiding Principles for Natural History Research...
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...
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...