Roche Imposes New Dosing Restrictions on Elevidys for Non-Ambulatory DMD Patients
Swiss pharmaceutical giant Roche AG (SWX: ROG, OTCMKTS: RHHBY) announced immediate new dosing restrictions for...
Rare / orphan disease drugs
Sanofi’s Rilzabrutinib Shows Promise in IgG4-Related Disease Trial
French pharmaceutical giant Sanofi (NASDAQ: SNY) announced positive results from a Phase II study of...
Biogen Launches Qalsody in China for SOD1-ALS Treatment
US-based Biogen (NASDAQ: BIIB) officially launched Qalsody (tofersen) in China this week. The drug, the...
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)...
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)...