NMPA CDE Launches “Caring Program – Extension” Pilot – Accelerates Rare Disease Drug Development Pathway
The Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) released the...
Rare / orphan disease drugs
Bio-Thera’s BAT4406F BLA Accepted by NMPA – Next‑Gen Anti‑CD20 mAb for Rare Disease NMOSD
Bio-Thera Solutions Inc. (SHA: 688177) announced that China’s National Medical Products Administration (NMPA) has accepted the...
FDA Issues Draft Guidance on Plausible Mechanism Framework – Accelerates Approval Pathway for Individualized Genetic Therapies
The U.S. Food and Drug Administration (FDA) released a draft guidance titled “Considerations for the...
Mabworks’ Obexelimab β Wins NMPA Approval as First Domestic NMOSD Therapy
Beijing Mabworks Biotech Co., Ltd. announced that the National Medical Products Administration (NMPA) has approved...
Amgen’s UPLIZNA Wins European Approval for Generalized Myasthenia Gravis
Amgen Inc. (NASDAQ: AMGN) announced that the European Commission (EC) has approved UPLIZNA (inebilizumab) as an...
Sanofi Invests $30 Million in GluBio’s Sickle Cell TPD Programs
GluBio Pharmaceutical Co., Ltd., a Zhejiang-based molecular glue targeted protein degradation (TPD) specialist, announced a...
Takeda’s Oveporexton Wins FDA Priority Review for Narcolepsy Type 1, Potential First Orexin Agonist
Takeda Pharmaceutical Company Limited (TYO: 4502, NYSE: TAK) announced that the U.S. Food and Drug Administration (FDA)...
Sanofi’s Wayrilz Wins FDA Breakthrough and Japan Orphan Designation for Rare Blood Disorder
Sanofi (NASDAQ: SNY) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy...
Pfizer’s Hympavzi Wins FDA Priority Review for Pediatric Hemophilia Expansion
Pfizer Inc. (NYSE: PFE) announced that the U.S. Food and Drug Administration (FDA) has accepted...
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...
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...