AstraZeneca’s Ultomiris Faces FDA Rejection for NMOSD Indication Extension
UK pharmaceutical company AstraZeneca (AZ; NASDAQ: AZN) has received a rejection from the US Food...
Rare / orphan disease drugs
Genentech Files for FDA Approval of Crovalimab for PNH, Challenging Soliris
Swiss pharmaceutical giant Roche’s subsidiary, Genentech (SWX: ROG), has submitted its anti-C5 investigational drug, crovalimab,...
Novo Nordisk’s Esperoct on Track for Priority Review in Hemophilia A Treatment
The Center for Drug Evaluation (CDE) website has indicated that Denmark-based Novo Nordisk’s (NYSE: NVO)...
Amgen and Horizon Therapeutics Reach Agreement with FTC to Proceed with Acquisition
Amgen (NASDAQ: AMGN) and Horizon Therapeutics (NASDAQ: HZNP) have announced a consent order agreement with...
AstraZeneca’s Soliris Gains Approval for Pediatric Generalized Myasthenia Gravis in Japan
UK pharmaceutical company AstraZeneca (AZ; NASDAQ: AZN) announced last week that it has received an...
Roche’s Evrysdi Receives EC Approval for Expanded Indication in Infants with SMA
Swiss pharmaceutical giant Roche (SWX: ROG) has received regulatory approval from the European Commission (EC)...
Cordavis Launches First Biosimilar: Novartis’ Hyrimoz to Compete with Humira
Swiss pharmaceutical giant Novartis (NYSE: NVS) unit Sandoz’s biosimilar product, Hyrimoz (adalimumab), which references AbbVie’s...
Auzone Biological Technology Gets FDA Approval for Edaravone Oral Preparations in ALS
Suzhou Auzone Biological Technology Co., Ltd. has received approval from the US Food and Drug...
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...
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...