Novartis Remibrutinib Beats Teriflunomide in Phase III REMODEL Trials for Relapsing Multiple Sclerosis
Novartis (NYSE: NVS) announced positive topline results from its Phase III REMODEL-1/-2 clinical trials of...
Rare / orphan disease drugs
Shanghai RAAS’s SR604 Enters NMPA CDE Rare Disease Encouragement Program for Hemophilia and Factor VII Deficiency Treatment
China’s Shanghai RAAS (SHE: 002252) announced that its candidate SR604 has been included by the...
GluBio’s GLB‑005 WIZ Degrader Enters NMPA Rare Disease Pilot for β‑Thalassemia
GluBio Pharmaceuticals announced that GLB‑005, its self‑developed widely interspaced zinc finger (WIZ) molecular glue degrader...
Roivant’s Lisraya Wins FDA Approval as First Targeted Therapy for Dermatomyositis
Roivant (NASDAQ: ROIV) announced that the US Food and Drug Administration (FDA) has approved Lisraya...
FDA Approves J&J’s IMAAVY for Warm Autoimmune Hemolytic Anemia, Marking Second Indication for Nipocalimab
Johnson & Johnson (J&J, NYSE: JNJ) announced that the US FDA has approved the second...
Ultomiris Wins NMPA Approval for PNH in China, Bringing Long‑Acting Ravulizumab C5 Inhibitor to Adult and Pediatric Patients
Ultomiris (ravulizumab injection), a long‑acting C5 complement inhibitor under AstraZeneca’s (NYSE: AZN) Rare Disease business,...
Hengrui Wins NMPA Approval for HRS‑5965 – Complement Factor B Inhibitor for Complement Inhibitor‑Naive PNH Patients
Jiangsu Hengrui Pharmaceuticals Co., Ltd. (SHA: 600276; HKG: 1276) announced that China’s National Medical Products...
LEO Pharma to Acquire Global Rights to Dersimelagon From Tanabe – Late‑Stage Oral Therapy for Rare Protoporphyrias Nears FDA Decision
LEO Pharma A/S announced that it has entered into an agreement with Tanabe Pharmaceutical to...
CANbridge Pharmaceuticals’ Livmarli Granted NHIA Reimbursement Approval in Taiwan – IBAT Inhibitor Coverage for ALGS and PFIC Takes Effect September 1
CANbridge Pharmaceuticals Inc. (HKG: 1228) announced that its rare disease drug Livmarli (Maralixibat Chloride Oral...
Yiling Pharmaceutical Files IND in China for LB220-D08, a First-In-Class Factor B Inhibitor for aHUS
Yiling Pharmaceutical (SHE: 002603) announced that it has submitted an Investigational New Drug (IND) application...
CDE Adds Two New Drugs to Rare Disease “Caring Program – Extension” Pilot – SZJ0107 and JAB-8263 Included
According to the latest announcement from the Center for Drug Evaluation (CDE) of the National...
Jazz Pharmaceuticals to Acquire Actio Biosciences for Up to USD 1.32 Billion, Gaining First‑in‑Class KCNT1 Ion Channel Inhibitor ABS‑1230 for Ultra‑Rare Epilepsy
Jazz Pharmaceuticals (NASDAQ: JAZZ) announced that it will acquire Actio Biosciences, a privately held biopharmaceutical...
Takeda Expands Revestive Approval in China with New 1.25 mg Specification for Pediatric Short Bowel Syndrome Patients as Young as 4 Months
Takeda Pharmaceutical Company Limited (TYO: 4502; NYSE: TAK) announced that China’s National Medical Products Administration...
Takeda Receives FDA Approval for ORZEYFUL (oveporexton), First Oral OX2R Agonist for Narcolepsy Type 1 in Adults
Takeda Pharmaceutical Company Limited (TYO: 4502; NYSE: TAK) announced that the U.S. Food and Drug...
Zai Lab’s Zocilurtatug Pelitecan Receives FDA Orphan Drug Designation for Neuroendocrine Carcinoma
Zai Lab (NASDAQ: ZLAB; HKG: 9688) announced that the US Food and Drug Administration (FDA)...
AstraZeneca’s Ultomiris Fails Primary Endpoint in Adult HSCT-TMA Phase III Trial Despite Positive Pediatric Data
AstraZeneca plc (NYSE: AZN) announced that its ALXN1210-TMA-313 Phase III clinical trial failed to meet...
Hengrui’s Tissue Factor-Targeting ADC SHR-4375 Receives FDA Orphan Drug Designation for Pancreatic Cancer
Jiangsu Hengrui Pharmaceuticals Co., Ltd. (SHA: 600276; HKG: 1276) announced that its innovative antibody-drug conjugate...
Kanghong Pharmaceutical Secures NMPA Approval for KHN921 Gene Therapy in MYBPC3-Related Hypertrophic Cardiomyopathy Following U.S. FDA Clearance
Kanghong Pharmaceutical Group Co., Ltd. (SHE: 002773) announced on July 23, 2026 that China’s National...
Haisco Pharma Secures NMPA Approval for HSK39297 – First Oral CFB Inhibitor for Paroxysmal Nocturnal Hemoglobinuria in China
Haisco Pharmaceutical Group Co., Ltd. (SHE: 002653) announced that China’s National Medical Products Administration (NMPA)...
Takeda Secures World’s First NMPA Approval for Orexatone – Novel Oral OX2R Agonist for Type 1 Narcolepsy
Takeda Pharmaceutical Company Limited (TYO: 4502; NYSE: TAK) announced that China’s National Medical Products Administration...