Novartis Wins CHMP Positive Opinion for Itvisma (Intrathecal Onasemnogene Abeparvovec) in 5q Spinal Muscular Atrophy Across All Ages in EU
Novartis AG (NYSE: NVS) announced that the Committee for Medicinal Products for Human Use (CHMP)...
Rare / orphan disease drugs
Boehringer Ingelheim Launches Pulmonary Fibrosis Care Initiative in China with CNR News, Tencent Medipedia, and Patient Groups to Combat Underdiagnosed Fatal Lung Disease
Boehringer Ingelheim, the German pharmaceutical giant, officially launched the Pulmonary Fibrosis Care Initiative late last...
Insilico Medicine Advances AI-Designed PRMT5 Inhibitor ISM0387 for Glioblastoma with Sub-12-Month Discovery Timeline
Insilico Medicine (HKG: 3696), a China-based artificial intelligence (AI) drug discovery platform, announced the selection...
Roche’s Fenebrutinib Slashes MS Relapse Rates by Over 50% in Phase III Trials
Roche AG (SWX: ROP, OTCMKTS: RHHBY) announced that its investigational non-covalent Bruton’s tyrosine kinase (BTK)...
Novo Nordisk’s Oral Etavopivat Meets Primary Endpoints in Phase III HIBISCUS Trial for Sickle Cell Disease; Regulatory Filing Planned for H2 2026
Novo Nordisk A/S (NYSE: NVO) announced positive topline results from the Phase III HIBISCUS trial...
Kanghong Pharmaceutical Receives FDA Clearance for KHN921 Gene Therapy Trial in MYBPC3-Associated Hypertrophic Cardiomyopathy – Precision AAV Vector Targets Myocardial Tissue
Chengdu Kanghong Pharmaceutical Group Co., Ltd. (SHE: 002773) announced it has received Investigational New Drug...
GSK Acquires 35Pharma for $950 Million to Secure Best-in-Class Pulmonary Arterial Hypertension Therapy HS235
GlaxoSmithKline plc (GSK, NYSE: GSK) has completed its acquisition of 35Pharma Inc., a Canadian biopharmaceutical...
UCB Secures EU Approval for Kygevvi as First-Ever Treatment for Ultra-Rare TK2 Deficiency
UCB (EBR: UCB) announced that the European Commission (EC) has granted marketing approval under exceptional...
Neurocrine Biosciences to Acquire Soleno Therapeutics for $2.9 Billion, Adding Three First-in-Class Commercial Assets
Neurocrine Biosciences, Inc. (NASDAQ: NBIX) has announced a definitive agreement to acquire fellow U.S. biopharmaceutical...
Amgen’s Subcutaneous Tepezza via On-Body Injector Meets Phase III Endpoint in Thyroid Eye Disease with 77% Proptosis Response
Amgen Inc. (NASDAQ: AMGN) announced positive top-line results from a Phase III clinical trial evaluating...
Rui Therapeutics Secures NMPA Approval for KN5501 CAR-NK Therapy in Autoimmune Diseases
Rui Therapeutics, a China-based cell therapy developer, announced it has received approval from the National...
Changchun High & New Receives NMPA Approval for GenSci161 Clinical Study in Endometriosis and Hidradenitis Suppurativa
Changchun High & New Technology Industries (Group) Inc. (SHE: 000661) announced it has received approval...
Biogen Secures FDA Approval for High-Dose Spinraza Regimen in Spinal Muscular Atrophy
Biogen Inc. (NASDAQ: BIIB) announced that the U.S. Food and Drug Administration (FDA) has approved...
Abbisko Therapeutics Secures EMA Orphan Drug Designation for Irpagratinib in Hepatocellular Carcinoma
Abbisko Therapeutics Co., Ltd. (HKG: 2256) announced that the European Medicines Agency (EMA) has granted...
Abbisko Therapeutics Secures FDA Clearance for ABSK061 Pediatric Achondroplasia Trial, Advancing Oral FGFR2/3 Inhibitor
Abbisko Therapeutics Co., Ltd. (HKG: 2256) has announced FDA clinical trial approval for its investigational...
Hansoh Pharma’s Uplizna Gains Third NMPA Approval in China for Generalized Myasthenia Gravis, Expanding CD19 Antibody Franchise
Hansoh Pharmaceutical Group Co., Ltd. (HKG: 3692) announced that China’s National Medical Products Administration (NMPA)...
UCB’s Bimzelx Wins NMPA Approval for Two New Indications – IL-17A/F Dual-Target Antibody Expands into Psoriasis and Hidradenitis Suppurativa
UCB (EBR: UCB) announced National Medical Products Administration (NMPA) approval for Bimzelx (bimekizumab) in two...
FDA Approves Rocket Pharma’s Kresladi – First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I
The U.S. Food and Drug Administration (FDA) this week approved Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel),...
Pulmongene’s PMG1015 Wins FDA Fast Track Designation – First-in-Class AREG Antibody Targets Idiopathic Pulmonary Fibrosis
Pulmongene (Beijing) Ltd. announced that PMG1015 received Fast Track Designation (FTD) from the U.S. Food...
Changchun High-Tech Wins FDA Orphan Drug Designation for GenSci128 – TP53 Y220C Reactivator Targets Gastric Cancer
Changchun High & New Technology Industries (Group) Inc. (SHE: 000661) announced that GenSci128 received Orphan...