Sinogen’s SalMet-Vec Receives FDA Orphan Drug Designation for Pancreatic Cancer
China’s oncology specialist Guangzhou Sinogen Pharmaceutical Co., Ltd. has announced receiving Orphan Drug Designation (ODD)...
Rare / orphan disease drugs
FDA Grants Priority Review to Johnson & Johnson’s Nipocalimab for Myasthenia Gravis
US-based Johnson & Johnson (J&J, NYSE: JNJ) has announced that the US FDA has granted...
Alchemab Therapeutics Partners with Eli Lilly to Discover ALS Treatments
UK-based Alchemab Therapeutics has formed a strategic partnership with US major Eli Lilly & Co....
HuidaGene Therapeutics Receives EMA Orphan Designation for HG004 Gene Therapy
Shanghai-based genome medicines specialist HuidaGene Therapeutics has announced a positive opinion from the European Medicines...
Takeda and Fudan University’s IMI Launch AI-Based Rare Disease Screening and Diagnosis Project
Japan-headquartered Takeda (TYO: 4502) and the Intelligent Medicine Institute (IMI) at Fudan University have jointly...
Novartis Announces Positive Results from Phase III STEER Study on SMA Treatment
Swiss pharmaceutical giant Novartis (NYSE: NVS) has announced positive topline results from the Phase III...
MicuRx Pharmaceuticals’ MRX-5 Receives FDA Orphan Drug Designation for NTM Infections
Sino-US firm MicuRx Pharmaceuticals Inc. has announced that the US Food and Drug Administration (FDA)...
argenx’s Vygart Hytrulo Approved in Japan for Chronic Inflammatory Demyelinating Polyneuropathy
Holland-based argenx SE (NASDAQ: ARGX) has announced receiving another indication approval from Japan’s Ministry of...
Sanofi and Corxel Pharmaceuticals Partner for Aficamten in Greater China Rights
Global healthcare leader Sanofi (NASDAQ: SNY) and China-based Corxel Pharmaceuticals (CORXEL), formerly known as Ji...
Sino Biopharmaceutical Ltd. Files for Benmelstobart and Anlotinib Combination Therapy in ASPS Treatment
China-based Sino Biopharmaceutical Ltd (HKG: 1177) is poised to make another indication approval filing with...
AstraZeneca’s ALXN2350 Selected for CDE’s Rare Disease Drug Development Program
The Center for Drug Evaluation (CDE) in China has announced its intention to include AstraZeneca’s...
Vcanbio’s VUM02 Secures NMPA Approval and FDA ODD for Cirrhosis and aGvHD
China-based Vcanbio Cell & Gene Engineering Corp., Ltd (SHA: 600645) has announced significant progress for...
European CHMP Supports Conditional Approval of MSD’s Welireg for VHL Disease and RCC
Merck, Sharp & Dohme (MSD; NYSE: MRK) has announced that the European Medicines Agency’s Committee...
EMA’s CHMP Recommends Approval of Bayer’s Acoramidis for ATTR-CM Treatment
Bayer’s (ETR: BAYN) acoramidis has received a positive recommendation from the Committee for Medicinal Products...
Wepon Medical’s WP107 for Myasthenia Gravis Accepted for FDA Review
Wepon Medical Holding Group Co., Ltd. (SHE: 002082) has announced that the US Food and...
HuidaGene Therapeutics Launches MUSCLE Study for CRISPR Therapy in Duchenne Muscular Dystrophy
Shanghai-based genome medicines specialist HuidaGene Therapeutics has announced the initiation of the MUSCLE study for...
Shanghai Pharmaceuticals’ SRD4610 Receives FDA Orphan Drug Designation for ALS Treatment
China’s Shanghai Pharmaceuticals (SPH; HKG: 2607; SHA: 601607) has announced that the US Food and...
Sperogenix Therapeutics’ Agamree Receives NMPA Approval for Duchenne Muscular Dystrophy Treatment
China-based rare disease specialist Sperogenix Therapeutics Limited has announced receiving marketing approval from the National...
HuidaGene Therapeutics Initiates HERO Trial for RNA-Editing Therapy HG204 in MECP2 Duplication Syndrome
Shanghai-based HuidaGene Therapeutics has announced the first subject dosing of the HERO clinical trial, which...
Novartis’ Fabhalta Demonstrates Positive Results in APPULSE-PNH Phase IIIb Study
Swiss pharmaceutical major Novartis (NYSE: NVS) has released positive topline results from the APPULSE-PNH Phase...