Sanofi’s Rilzabrutinib Granted Orphan Drug Designation for Sickle Cell Disease
France-based Sanofi (NASDAQ: SNY) announced that it has received an additional Orphan Drug Designation (ODD)...
Rare / orphan disease drugs
Sanofi to Acquire Blueprint Medicines for $9.1 Billion to Expand Rare Disease Pipeline
French pharmaceutical giant Sanofi (NASDAQ: SNY) announced plans to acquire US-based Blueprint Medicines Corporation for...
R&B Biotech Receives FDA Approval for ZS805 Gene Therapy in Fabry Disease
Sichuan R&B Biotech, a China-based biotech firm, announced receiving clinical approval from the US Food...
Novartis’ Fabhalta Approved for Previously Treated PNH Patients in China
The China unit of Swiss pharmaceutical giant Novartis (NYSE: NVS) announced that the National Medical...
RemeGen’s Telitacicept Approved for Myasthenia Gravis in China
China-based pharmaceutical firm RemeGen (HKG: 9995) announced the third indication approval from the National Medical...
Mabworks’ MIL62 Gains NMPA Review for NMOSD Treatment
China-based Beijing Mabworks Biotech Co., Ltd announced that its New Drug Application (NDA) for MIL62,...
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...
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...