CANbridge’s Maralixibat Receives Priority Review for PFIC Treatment in China
China-based rare disease specialist CANbridge Pharmaceuticals Inc. (HKG: 1228) has announced that a New Drug...
Rare / orphan disease drugs
Roche’s Ocrevus Demonstrates Long-Term Efficacy in Multiple Sclerosis Treatment
Swiss pharmaceutical giant Roche (SWX: ROG) has presented compelling long-term efficacy data for its selective...
Kintor Pharmaceutical Secures NMPA Approval for Phase II Study of GT1708F in Idiopathic Pulmonary Fibrosis
China-based Kintor Pharmaceutical Ltd (HKG: 9939) has announced that it has received approval from the...
Gritgen Therapeutics Commences Operations of Commercial GMP Facilities in Suzhou
Suzhou-based gene therapy specialist, Gritgen Therapeutics Co., Ltd, has announced the validation completion and initiation...
GenFleet Therapeutics’ CDK9 Inhibitor SLS009 Earns Orphan Drug Status for AML
Shanghai-based biotechnology company GenFleet Therapeutics has announced that its CDK9 inhibitor, SLS009 (GFH009), has received...
Shanghai BDgene’s BD112 Earns Orphan Drug Designation from US FDA for Huntington’s Disease
Shanghai BDgene Technology Co., Ltd, a specialist in gene therapy, has announced that its drug...
Alphamab Oncology Enters Licensing Agreement with Grand Life Sciences for KN057 in Greater China
China-based Alphamab Oncology (HKG: 9966) has announced a licensing agreement with fellow Chinese firm Grand...
Sanofi’s Nexviazyme Approved in China as Enzyme Replacement Therapy for Pompe Disease
Sanofi’s (NASDAQ: SNY) next-generation enzyme replacement therapy (ERT), Nexviazyme (avalglucosidase alfa), has received marketing approval...
Amicus Therapeutics Secures FDA Approval for Pombiliti and Opfolda Combination Therapy
Amicus Therapeutics (NASDAQ: FOLD), headquartered in the United States, has announced that it has received...
Novo Nordisk and Evotec Launch LAB eN² to Accelerate Drug Discovery for Rare Diseases
Global healthcare company Novo Nordisk (NYSE: NVO) and drug discovery alliance Evotec (NASDAQ: EVO) have...
Sanofi’s Altuviiio Receives Marketing Approval from Japan’s MHLW for Hemophilia A Treatment
French pharmaceutical giant Sanofi (NASDAQ: SNY) has announced that it has received marketing approval from...
Novartis’ Lutathera Meets Primary Endpoint in Phase III Trial for GEP-NETs
Swiss pharmaceutical giant Novartis (NYSE: NVS) has announced that a Phase III trial for its...
EU Chamber of Commerce in China Releases Position Paper, Addresses Pharma and Healthcare
The EU Chamber of Commerce in China has released its bi-annual “European Business in China...
Organon’s Liptruzet Approved in China for Hypercholesterolemia Treatment
US-based Organon (NYSE: OGN) has announced that it has received market approval from the National...
China’s NHC Expands Rare Disease List, Includes 21 Cancer Types
The National Health Commission (NHC) of China has released an updated national list of rare...
Caliway Biopharmaceuticals’ CBL-514 Achieves Milestones in Dercum’s Disease Trial
Taiwan-based Caliway Biopharmaceuticals, a specialist in medical aesthetics and inflammatory medicine, has announced that its...
AstraZeneca’s Fasenra Shows Non-Inferior Efficacy to Nucala in EGPA: Phase III Results
UK-based pharmaceutical giant AstraZeneca (AZ; NASDAQ: AZN) has unveiled the results of a Phase III...
NMPA Pilots Priority Review for Rare Disease Formula Foods, Aims to Reduce Approval Time
The National Medical Products Administration (NMPA) has initiated a pilot program to implement “Priority review...
Grand Pharmaceutical Receives NMPA Approval for Generic Carbaglu for Hyperammonemia Treatment
China-based Grand Pharmaceutical Group Limited (HKG: 0512) has announced that it has received marketing approval...
CDE Indicates BTD Status for Visirna’s VSA001, Sperogenix’s Vamorolone, and Zai Lab’s Efgartigimod
The Center for Drug Evaluation (CDE) website has indicated that Visirna Therapeutics’ novel siRNA therapy...