Fosun Pharma’s HLX22 Granted Orphan Drug Designation by EU Commission
China-based Shanghai Fosun Pharmaceutical (Group) Co., Ltd (SHA: 600196, HKG: 2196) announced that it has...
Rare / orphan disease drugs
Merck’s Welireg Launched in China for Rare Genetic Disorder Treatment
US-based Merck, Sharp & Dohme (MSD; NYSE: MRK) announced the commercial launch of its hypoxia-inducible...
AccurEdit Therapeutics’ ART001 Receives FDA RMAT Designation for ATTR
China-based gene editing therapy specialist AccurEdit Therapeutics (Suzhou) Co., Ltd announced receiving Regenerative Medicine Advanced...
Pulnovo Medical Raises Tens of Millions in New Financing Round
Wuxi-based Pulnovo Medical Limited, a developer of medical devices for hypertension and heart failure, announced...
CANbridge’s Velaglucerase β Approved by NMPA for Gaucher’s Disease Long-Term Therapy
China-based CANbridge Pharmaceuticals Inc. (HKG: 1228) announced that the National Medical Products Administration (NMPA) has...
Tenacia Partners with TSH Biopharm for Ztalmy Commercialization in Taiwan
Tenacia Biotechnology, a central nervous system (CNS) specialist backed by Bain Capital, has entered into...
UCB Gets Japanese Approval for New Administration Routes of Rystiggo in gMG
Belgium-based biopharma UCB (EBR: UCB) announced that it has received marketing approval from Japan’s Pharmaceuticals and...
IASO Biotherapeutics’ Fucaso Granted Orphan Drug Designation by SFDA for Multiple Myeloma
China-based IASO Biotherapeutics announced that the Saudi Food and Drug Authority (SFDA) has granted Orphan...
Alchemab Therapeutics Licenses ATLX-1282 to Eli Lilly for ALS Treatment
UK-based biotech Alchemab Therapeutics Ltd entered into a licensing agreement with US major Eli Lilly...
Pfizer’s Q1 2025 Results: Revenues Down 6% YOY Amid Paxlovid, IRA Impact
US-based Pfizer Inc. (NYSE: PFE) released its Q1 2025 financial results, showing a -6% year-on-year...
AstraZeneca’s Ultomiris Approved in China for Generalized Myasthenia Gravis
UK-based pharmaceutical giant AstraZeneca (NASDAQ: AZN) has announced that the National Medical Products Administration (NMPA)...
Belief BioMed Gains NMPA Clinical Trial Approval for DMD Gene Therapy BBM-D101
China-based gene therapy specialist Belief BioMed Inc. (BBM) has announced that it has received clinical...
Biogen’s High-Dose Spinraza Regimen Accepted for CDE Review in China
US-based Biogen Inc. (NASDAQ: BIIB) announced that the Center for Drug Evaluation (CDE) in China...
Takeda’s Adzynma, BeiGene’s Sonrotoclax, and Avistone’s Andatinib Set for Priority Review in China
The website of China’s Center for Drug Evaluation (CDE) indicates that Takeda’s (TYO: 4502) Adzynma...
Belief BioMed and Takeda Win NMPA Approval for China’s First Hemophilia B Gene Therapy
China-based gene therapy specialist Belief BioMed Inc. and Takeda’s (NYSE: TAK, TYO: 4502) China unit...
HemaCell’s XJ-MK-002 Gains FDA Orphan Drug Designation for Giant Platelet Syndrome
China-based Suzhou Xueji Biotechnology Co., Ltd (HemaCell), a pioneer in platelet regeneration-focused cell therapy, announced...
Sanofi’s Rilzabrutinib Granted Orphan Drug Designation for Rare Diseases
Sanofi (EPA: SAN, NASDAQ: SNY) announced that it has received Orphan Drug Designation (ODD) from...
Sarepta and Roche Seek to Resume Elevidys Trials After EMA Halt
The European Medicines Agency (EMA) may lift the temporary hold on certain Phase III studies...
Hengrui Pharmaceuticals Secures Clinical Trial Approval for HRS-9813 in IPF
Hengrui Pharmaceuticals (SHA: 600276) announced on April 1, 2025, that its HRS-9813 capsule has received...
Sanofi’s Qfitlia Approved by FDA for Hemophilia A and B Prophylaxis
France-based Sanofi (EPA: SAN, NASDAQ: SNY) has announced receiving marketing approval from the US Food...