KPC Pharmaceuticals Gets NMPA Green Light for Clinical Trial on Familial Mediterranean Fever Treatment
Yunnan-based KPC Pharmaceuticals Inc., (SHA: 600422) has announced that it has received clinical trial approval...
Rare / orphan disease drugs
Shanghai’s 9th People’s Hospital Launches Groundbreaking Gene Therapy Trial for Congenital Deafness
In a medical first, Shanghai’s 9th People’s Hospital has initiated the world’s inaugural clinical trial...
Ipsen Secures NMPA Approval for Somatuline in Advanced Neuroendocrine Tumors
Ipsen (EPA: IPN; OTCMKTS: IPSEY), a France-based biopharmaceutical company, has received new indication approval from...
Alexion’s Voydeya Earns FDA Nod as Add-on Therapy for PNH-Associated Extravascular Hemolysis
Alexion, the rare disease unit of AstraZeneca (AZ; NASDAQ: AZN) based in the UK, has...
J&J’s Nipocalimab Fast-Tracked by FDA for Reducing FNAIT Risk in Pregnant Adults
The US Food and Drug Administration (FDA) has accepted Johnson & Johnson’s (J&J; NYSE: JNJ)...
Takeda Gains MHLW Approval for Adzynma, Targeting Rare Blood Disorder cTTP
Takeda Pharmaceutical Company (TYO: 4502) has secured regulatory approval from Japan’s Ministry of Health, Labour...
Alexion’s Ultomiris Secures FDA Approval for Anti-AQP4 Positive NMOSD Treatment
AstraZeneca’s (AZ; NASDAQ: AZN) rare disease unit, Alexion, has registered its biologic Ultomiris (ravulizumab) with...
Jiangsu Hengrui’s Generic Version of Tepadina Approved for Transplantation Conditioning in China
Jiangsu Hengrui Pharmaceuticals Co., Ltd (SHA: 600276), a leading pharmaceutical company based in China, has...
EMA Panel Backs Novartis’s Fabhalta for PNH Treatment, Awaits EC Decision
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)...
iRegene’s NouvSight001 Gains Orphan Drug Designation for Retinitis Pigmentosa Treatment
Wuhan-based cell therapy innovator iRegene has secured Orphan Drug Designation (ODD) from the U.S. FDA...
Shanghai BDgene Partners with SPH RD to Advance Gene Therapy Development
Shanghai BDgene Technology Co., Ltd., a China-based specialist in gene therapy, has entered into a...
Boehringer Ingelheim’s Spevigo Receives NMPA Approval for GPP Treatment in China
German pharmaceutical giant Boehringer Ingelheim (BI) has announced that it has received marketing approval from...
Novartis Gene Therapy Zolgensma Shows Continued Benefits in Broader SMA Patient Group
Swiss pharmaceutical giant Novartis (NYSE: NVS) has presented Phase III data this week, demonstrating that...
BMS Reports Positive Long-Term Data for Zeposia in Multiple Sclerosis Trials
Bristol Myers Squibb (BMS; NYSE: BMY) has unveiled results from several Phase III trials for...
Merck’s Mavenclad Demonstrates Lasting Efficacy in RMS Patients, New Data Show
Merck KGaA (ETR: MRK), a German multinational pharmaceutical, chemical, and life sciences company, has this...
Hainan Poly Pharm Secures Tentative FDA Approval for Generic Version of UCB’s Briviact
Hainan Poly Pharm Co., Ltd (SHE: 300630), a Chinese pharmaceutical company, has announced that it...
CANbridge Pharmaceuticals Partners with Peking Union Medical College to Advance Rare Disease Research
CANbridge Pharmaceuticals Inc. (HKG: 1228), a specialist in rare diseases based in China, has announced...
Jiangsu Nhwa Secures Exclusive Rights to Teva’s Austedo in China with $30 Million Deal
Jiangsu Nhwa Pharmaceutical Co., Ltd. (SHE: 002262), based in China, has entered into a cooperation...
Immune-Onc Therapeutics’ IO-202 Earns Orphan Drug Designation for CMML from US FDA
Immune-Onc Therapeutics, the Hangzhou-based subsidiary of US company Immune-Onc Therapeutics Inc., has announced that it...
MSD’s Belzutifan and Sperogenix’s Vamorolone Targeted for Priority Review by China’s CDE
The Center for Drug Evaluation (CDE) website has indicated that Merck, Sharp & Dohme’s (MSD;...