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 for Niemann-Pick disease type C (NPC), a ... Read More
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 in an all-cash transaction valued at $466 ... Read More
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. Food and Drug Administration (FDA) approval of ... Read More
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 for rare endocrine diseases, in a deal ... Read More
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 Series B financing round. The round was ... Read More
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 the efficacy of givosiran in treating acute ... Read More
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 leader in rare disease treatments. This proposal, ... Read More
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 acquisition of Swedish pharmaceutical company Wilson Therapeutics ... Read More
Mallinckrodt to acquire Sucampo Pharmaceuticals in $1.2bn deal
In a strategic move to broaden its pharmaceutical offerings, UK-based Mallinckrodt has announced the acquisition of Sucampo Pharmaceuticals, including its commercial and developmental assets, for ... Read More