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...
Rare / orphan disease drugs
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,...
Amgen’s Uplizna Scores Third FDA Indication in Myasthenia Gravis
Amgen Inc. (NASDAQ: AMGN) announced that the U.S. FDA approved Uplizna (inebilizumab-cdon) for adult patients...
Sanofi’s Qfitlia Wins China Approval as First Hemophilia siRNA Therapy
Sanofi (NASDAQ: SNY) announced that Qfitlia (Fitusiran Sodium Injection), the world’s first and only siRNA‑based...
FDA Waskyra WAS Gene Therapy First Cell-Based
The US Food and Drug Administration (FDA) approved Waskyra (etuvetidigene autotemcel) as the first cell‑based...
Lynk Pharma LNK01006 FDA Approval Targets TYK2 for CNS Diseases
Lynk Pharmaceuticals Co., Ltd. announced that its self‑developed TYK2 allosteric inhibitor LNK01006 has received FDA...
Johnson & Johnson’s Nipocalimab Secures EU Approval for Myasthenia Gravis
Johnson & Johnson (J&J, NYSE: JNJ) announced that the European Commission has granted Marketing Authorisation...
iRegene’s NouvNeu004 Wins FDA Special Exemption for MSA Cell Therapy
iRegene Therapeutics announced that the U.S. FDA granted Special Exemption status to NouvNeu004 injection, approving...
Novartis’s Itvisma Wins FDA Approval as First Gene Therapy for SMA
Novartis AG (NYSE: NVS) announced that Itvisma (onasemnogene abeparvovec‑brve) received U.S. FDA approval for children...
Pfizer’s Hympavzi Wins China Approval as First Weekly Non‑Factor Hemophilia Therapy
Pfizer Inc. (NYSE: PFE) announced that Hympavzi (marstacimab), the world’s first non‑factor hemophilia therapy requiring...
FDA Issues Boxed Warning on Elevidys (delandistrogene moxeparvovec‑rokl) – Use Limited to Ambulatory DMD Patients Aged 4+
The U.S. Food and Drug Administration (FDA) announced a Boxed Warning for Elevidys, Sarepta Therapeutics’...
Zelgen Biopharma’s ZG006 (Alveltamig) Receives FDA Orphan‑Drug Designation for Neuroendocrine Carcinomas
Suzhou Zelgen Biopharmaceuticals Co., Ltd. (SHA: 688266) announced that its trispecific antibody ZG006 (alveltamig) has been...
FDA Announces New ‘Plausible‑Mechanism’ Pathway to Rapid Approvals for Rare Disease Treatments
The U.S. Food and Drug Administration (FDA) announced on Wednesday a landmark regulatory shift that could...
Humanwell Healthcare Secures NMPA Approval to Launch First Fixed‑Dose Combination for Pulmonary Arterial Hypertension
Humanwell Healthcare (Group) Co., Ltd (SHA: 600079) announced that its investigational compound HWH217 has received...
UCB Wins FDA Approval for KYGEVVI in Rare Mitochondrial Disease TK2d
UCB (EBR: UCB) announced that KYGEVVI received FDA approval for the treatment of adult and...
Jiangsu Hengrui Secures Priority Review for HRS‑5965 in China’s Rare‑Disease Portfolio
Jiangsu Hengrui Pharmaceuticals (SHA: 600276, HKG: 1276) announced that the National Medical Products Administration (NMPA)...