Nxera Pharma FY2025 Results Signal Pipeline Expansion
TOKYO, Feb. 13, 2026 — Nxera Pharma Co., Ltd. reported its consolidated financial and operational results for FY2025, outlining...
rare diseases
KalVista Showcases EKTERLY Data at AAAAI 2026 Meet
FRAMINGHAM, Mass. & SALISBURY, England, Feb. 12, 2026 — KalVista Pharmaceuticals announced the presentation of new clinical and real-world...
MIPLYFFA Real-World Data Strengthen NPC Treatment Impact
CELEBRATION, Fla., Feb. 4, 2026 —Zevra Therapeutics announced the presentation of positive new real-world data on MIPLYFFA® (arimoclomol) in...
Sensorion Secures €60M to Advance Gene Therapies for Hearing Loss
MONTPELLIER, France | January 28, 2026 — Sensorion, a clinical-stage biotechnology company focused on hearing loss disorders, announced a...
Italfarmaco–JCR Givinostat Deal Expands Rare Disease Pharma in Japan
MILAN, Italy & HYOGO, Japan | December 24, 2025 — Italfarmaco and JCR Pharmaceuticals have announced a landmark commercialisation...
France Grants Compassionate Access to Gene Therapy LUMEVOQ®
December 22, 2025 | Paris, France — GenSight Biologics announced that France’s medicines safety authority has granted Compassionate Use...
Sharp Therapeutics Strengthens Path to Gaucher Trials
December 19, 2025 | Pittsburgh, Pennsylvania and Toronto, Ontario — Sharp Therapeutics Corp. has announced an updated non-brokered private...
DAWNZERA Wins CHMP Backing for Preventing Hereditary Angioedema Attacks
Carlsbad, California, 14 November 2025 – Ionis Pharmaceuticals, Inc. and Otsuka Pharmaceutical Co., Ltd. announced that the Committee for...
KalVista Pharmaceuticals Highlights EKTERLY®’s Promise for Children and Strong Patient Satisfaction in HAE Treatment
Framingham, Massachusetts & Salisbury, England — November 6, 2025 : KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) presented new data at...
Gefurulimab Shows Early and Sustained Efficacy in gMG Patients
Cambridge, UK, 30 October 2025 – Alexion, AstraZeneca Rare Disease, today announced positive results from the global Phase III...