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...
Rare / orphan disease drugs
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...
Dewpoint Therapeutics Partners with Mitsubishi Tanabe Pharma to Develop ALS Treatment
US-based Dewpoint Therapeutics Inc. has entered into a strategic partnership with Japan-headquartered Mitsubishi Tanabe Pharma...
Ipsen’s Bylvay Receives NMPA Clearance for Treating Itching in PFIC Patients in China
France-based Ipsen (EPA: IPN) has announced that it has received marketing clearance from the National...
Tasly Pharma Receives NMPA Approval for Generic Lioresal (Baclofen) Oral Solution
China-based Tasly Pharma Co., Ltd (SHA: 600535) has announced that it has received marketing approval...
AccurEdit Therapeutics’ ART001 Shows Over 90% TTR Reduction in ATTR Amyloidosis Trial
Suzhou-based gene editing specialist AccurEdit Therapeutics has announced positive results from an ongoing investigator-initiated trial...
Novartis Secures Global Rights to PTC Therapeutics’ Huntington’s Disease Treatment PTC518
Swiss pharmaceutical giant Novartis (NYSE: NVS) has entered into a global license and collaboration agreement...
XellSmart Bio-Pharmaceutical Achieves Milestone with First Patient Enrollment in Global iPSC Clinical Study for ALS
XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd., in collaboration with Shanghai East Hospital (Tongji University Affiliated East...
CDE Expands Care Plan with Approvals for Ractigen’s RAG-17 and Vitalgen’s VGR-R01
In a swift succession of actions, China’s Center for Drug Evaluation (CDE) has added two...
Huadong Medicine’s Arcalyst Receives NMPA Approval for Cryopyrin-Associated Periodic Syndromes
China-based Huadong Medicine Co., Ltd (SHE: 000963) has announced receiving marketing approval from the National...
AstraZeneca’s ALXN1850 (Efzimfotase Alfa) Selected for China’s Rare Disease Care Plan
The Center for Drug Evaluation (CDE) in China has announced its intention to include AstraZeneca’s...
Merck’s Winrevair Shows Positive Results in Phase III ZENITH Study for PAH
US-based pharmaceutical giant Merck & Co., Inc. (NYSE: MRK) has announced positive interim analysis results...
Jiangsu Chia Tai Fenghai’s ALS Treatment FHND1002 Selected for CDE’s Patient-Centered Care Plan
China’s Center for Drug Evaluation (CDE) has announced its intention to include Jiangsu Chia Tai...