Beijing Aims to Establish Rare Disease Drug Security Pilot Zone with ‘White List’ System
Beijing is set to follow in the footsteps of Boao Lecheng with the release of...
Rare / orphan disease drugs
AstraZeneca’s Fasenra Secures FDA Nod for EGPA Treatment, Strengthening Rare Disease Portfolio
UK pharmaceutical giant AstraZeneca (NASDAQ: AZN, LON: AZN) has announced that the US Food and...
CDE Launches CARE Plan to Streamline Rare Disease Drug Development
The Center for Drug Evaluation has unveiled the ‘Pilot Work Plan for Patient-Centered Action for...
Suzhou GenAssist Launches First-of-Its-Kind Base Editing Drug Trial for Duchenne Muscular Dystrophy
Suzhou GenAssist Therapeutic Co., Ltd, a biopharmaceutical company based in China, has announced the commencement...
Jiangsu Hengrui’s ANGPTL3 Inhibitor SHR-1918 Earns Breakthrough Designation for HoFH Treatment
Jiangsu Hengrui Pharmaceuticals Co., Ltd (SHA: 600276), a leading pharmaceutical company in China, has announced...
Vcanbio’s Stem Cell Therapy VUM02 Gets NMPA Approval for Systemic Sclerosis Clinical Trial
Vcanbio Cell & Gene Engineering Corp., Ltd (SHA: 600645), a biotechnology company based in China,...
AccurEdit Therapeutics’ Gene-Editing Drug ART001 Clears FDA Hurdle for US Clinical Trials
SUZHOU—AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading Chinese specialist in gene editing therapies, has announced...
Tonghua Golden-Horse, UCB Seek China Approvals for Alzheimer’s, Myasthenia Gravis Drugs
China’s Tonghua Golden-Horse Pharmaceutical Industry Co. Ltd. and Belgium-based UCB (FRA: UNC) are seeking marketing...
China’s Supreme Court Deals Blow to Sanofi’s Aubagio, Confirms Patent Cancellation
BEIJING—In a recent development for the pharmaceutical industry, Sanofi’s multiple sclerosis therapy Aubagio (teriflunomide) has...
Gene Cradle Initiates First Regulatory Gene Therapy Study in China for Late-Onset Pompe Disease
Gene Cradle, a Beijing-based gene therapy specialist, has achieved a milestone by administering its GC301...
AstraZeneca’s Strensiq Approved for Rare Disease Therapy under Greater Bay Area’s Drug Access Policy
AstraZeneca (NASDAQ: AZN, LON: AZN) , a leading UK pharmaceutical company, has received approval for...
AstraZeneca’s Strensiq Gains Approval for Rare Disease Therapy in Guangdong Under “Hong Kong-Macau Drug and Device Access”
LONDON—AstraZeneca (NASDAQ: AZN, LON: AZN) , a leading UK-based pharmaceutical company, has announced that its...
Ascendis Pharma’s Hypoparathyroidism Therapy Yorvipath Wins FDA Nod, Fills Market Gap
This week, the U.S. Food and Drug Administration (FDA) granted market approval to Ascendis Pharma...
Zelgen Biopharmaceuticals’ ZG006 Earns Orphan Drug Designation from FDA for Small-Cell Lung Cancer
Suzhou Zelgen Biopharmaceuticals Co., Ltd (SHA: 688266), a biopharmaceutical company based in China, has announced...
Insilico Medicine’s ISM6331 Earns FDA Orphan Drug Designation for Mesothelioma Treatment
Insilico Medicine, a China-based biotechnology company at the forefront of generative artificial intelligence (AI), has...
BioNTech’s mRNA Cancer Immunotherapy BNT111 Achieves Primary Endpoint in Phase II Trial
Germany-based BioNTech SE (NASDAQ: BNTX) has announced that its mRNA cancer immunotherapy BNT111, in combination...
Organon’s Liptruzet Launches in China for Hypercholesterolemia Treatment
US pharmaceutical company Organon (NYSE: OGN) has announced the official market launch of its single-tablet...
Alphamab’s KN057 Earns FDA Orphan Drug Designation for Hemophilia B Treatment
Alphamab Oncology (HKG: 9966), a biopharmaceutical company based in China, has received another orphan drug...
Belief BioMed’s Hemophilia B Gene Therapy BBM-H901 Slated for Priority Review in China
The Center for Drug Evaluation (CDE) has indicated on its website that the market application...
Sinqi Pharmaceutical Gets NMPA Green Light for SQ-22031 Eye Drops to Treat Dry Eye Syndrome and Neurotrophic Keratitis
Shenyang Sinqi Pharmaceutical Co., Ltd (SHE: 300573), a pharmaceutical company based in China, has received...