Browsing Tag
rare disease treatment
11 posts
JR-446 developed by JCR Pharmaceuticals secures orphan drug designation from the European Commission for MPS IIIB
The European Commission has granted orphan drug status to JR-446 for MPS IIIB, supporting JCR Pharmaceuticals’ global rare disease expansion.
X4 Pharmaceuticals (NASDAQ: XFOR) gets FDA fast track for mavorixafor in chronic neutropenia
X4 Pharmaceuticals (XFOR) secures FDA Fast Track for mavorixafor in chronic neutropenia, targeting trial completion in 2025 and results in 2026.
Zevra Therapeutics’ MIPLYFFA receives FDA approval as first treatment for Niemann-Pick disease
Zevra Therapeutics Inc. has announced U.S. Food and Drug Administration (FDA) approval of MIPLYFFA (arimoclomol), a capsule-based treatment…
Cycle Pharmaceuticals proposes $466m acquisition of Vanda Pharmaceuticals
In a significant development within the pharmaceutical industry, UK-based Cycle Pharmaceuticals has offered to acquire Nasdaq-listed Vanda Pharmaceuticals…
FDA approves Italfarmaco’s Duvyzat for duchenne muscular dystrophy treatment
In a significant advancement for the treatment of Duchenne muscular dystrophy (DMD), Italfarmaco S.p.A. has announced the U.S.…
AstraZeneca to acquire Amolyt Pharma in $1.05bn deal to expand rare disease portfolio
AstraZeneca has announced a definitive agreement to acquire Amolyt Pharma, a clinical-stage biotechnology company focused on novel treatments…
Evox Therapeutics secures £35.5m in Series B to advance exosome therapeutics
Evox Therapeutics, an innovative biotechnology company spun out from Oxford University, has successfully raised £35.5 million in a…
Alnylam Pharmaceuticals completes patient enrollment for Phase 3 trial of givosiran in acute hepatic porphyrias
Alnylam Pharmaceuticals has announced the completion of patient enrollment for its pivotal phase 3 trial, ENVISION, which evaluates…
Takeda Pharmaceutical proposes $62bn acquisition of Shire to expand rare disease capabilities
Takeda Pharmaceutical, a major Japanese pharmaceutical company, has made a $62 billion offer to acquire Shire, an Ireland-based…
Alexion Pharmaceuticals to acquire Wilson Therapeutics for $855m to enhance rare disease treatment options
In a significant move to expand its rare disease drug portfolio, U.S.-based Alexion Pharmaceuticals has announced an all-cash…