Give Kids a Chance Act Extends FDA Pediatric Research Authority and PRV Program
The US Congress has introduced the Give Kids a Chance Act, legislation aimed at broadening...
Rare / orphan disease drugs
Venglustat Gaucher: Sanofi Meets Primary Endpoint in Phase 3 LEAP2MONO Study
Sanofi (NASDAQ: SNY) announced positive results from the LEAP2MONO phase 3 study (NCT05222906), showing venglustat met the...
Chiesi Fabry Drug Pegunigalsidase Alfa Wins CHMP Backing for Extended Dosing
Chiesi Group announced that the Committee for Medicinal Products for Human Use (CHMP) has recommended...
AstraZeneca’s Soliris Wins NMPA Approval for Pediatric Myasthenia Gravis
AstraZeneca plc (AZ, NASDAQ: AZN) announced that China’s National Medical Products Administration (NMPA) has approved Soliris...
Chiesi’s Raxone Begins LHON Treatment in China, First World‑Approved Therapy
Chiesi Group announced that the first patients have initiated treatment with Raxone (idebenone 150 mg) at...
Vitalgen’s VGN‑R08b Gene Therapy Earns FDA Orphan Drug Designation for Gaucher Disease
Shanghai Vitalgen BioPharma Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) has...
Hisoar Pharma Invests RMB 150 Million in Wanbangde’s WP205 for ALS, Securing 15 % Global Revenue Share
Zhejiang Hisoar Pharmaceutical Co., Ltd. (SHE: 002099) announced a strategic collaboration with Wanbangde New Building...
FDA Approves Zycubo as First Therapy for Pediatric Menkes Disease, Targeting Ultra‑Rare Copper Deficiency Disorder
The U.S. Food and Drug Administration (FDA) has approved Zycubo (copper histidinate) injection, the first...
BMS’s Camzyos Meets Primary Endpoint in Phase III SCOUT-HCM Study for Adolescent oHCM
Bristol-Myers Squibb (NYSE: BMY) announced that the SCOUT‑HCM Phase III study of Camzyos (mavacamten) met its primary...
Shanghai Pharma Receives NMPA Approval for Myasthenia Gravis Oral Solution
Shanghai Pharmaceuticals Holding Co., Ltd (SHA: 601607) announced that its subsidiary, Shanghai SPH Zhongxi Pharmaceutical...
NMPA Issues Priority Review Guidelines for Clinically Urgent Overseas Drugs
The National Medical Products Administration (NMPA) issued Announcement No. 3, 2026 on further optimizing the review...
XtalPi’s RTX-117 Wins NMPA IND for CMT Therapy
ReviR Therapeutics, an incubated company of XtalPi (HKG: 2228), announced that RTX-117 has received Investigational...
Merck’s Winrevair Wins China NMPA Approval for Pulmonary Arterial Hypertension
Merck Sharp & Dohme Inc. (MSD, NYSE: MRK) announced that Winrevair (sotatercept for injection), the world’s...
Sino Biopharmaceutical’s TDI01 Enrolls First IPF Patient in World’s First ROCK2 Inhibitor Phase III Trial
Sino Biopharmaceutical Limited (HKG: 1177) announced that TDI01, the world’s first highly selective ROCK2 inhibitor...
AstraZeneca and Ionis’ Eplontersen Wins China Approval for ATTRv-PN as First Gene-Silencing Therapy
AstraZeneca PLC (AZ, NASDAQ: AZN) and Ionis Pharmaceuticals (NASDAQ: IONS) announced that Wainua (eplontersen) has received approval...
Shionogi Acquires RADICAVA ALS Business for $2.5 Billion from Tanabe Pharma
Shionogi & Co., Ltd. (Japan) announced it will acquire 100% of the shares of a...
Gilead’s Seladelpar Wins China Hainan Approval for PBC Treatment
Gilead Sciences (NASDAQ: GILD) announced that Seladelpar (marketed as Livdelzi), a potent and selective PPARδ...
Novabio’s Treg Therapy NP001 Doses First ALS Patient in China Trial
Novabio Therapeutics, a China‑based clinical‑stage biotech, announced the successful completion of first patient dosing in...
Sanofi’s Tolebrutinib Fails Phase 3 PPMS Endpoint, Halting Registration Plans
Sanofi (NASDAQ: SNY) announced that the Phase 3 PERSEUS study (NCT04458051) of tolebrutinib in primary progressive...
Sobi Acquires Arthrosi for $1.5 Billion to Add Gout Drug to Portfolio
Swedish Orphan Biovitrum AB (Sobi) (STO: SOBI) announced a definitive agreement to acquire Arthrosi Therapeutics,...