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 rare and fatal neurodegenerative disease. This is the first FDA-approved treatment for NPC, which affects around 900 people in the U.S. and has no prior treatment options. MIPLYFFA is designed […]
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 million. Under this proposal, Cycle Pharmaceuticals plans to purchase shares of Vanda Pharmaceuticals at $8 per share, representing a substantial premium over recent closing prices. Financial Details and Premiums Offered […]
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 Duvyzat (givinostat), a novel histone deacetylase (HDAC) inhibitor, for patients aged 6 and older. This approval signifies a major milestone in the management of DMD, a rare and progressive neuromuscular […]
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 valued at up to $1.05 billion. This acquisition is poised to strengthen the late-stage pipeline of Alexion, AstraZeneca Rare Disease, particularly bolstering its bone metabolism franchise through the addition of […]
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 led by Redmile Group, with notable new investments from Google’s GV, Cowen Healthcare Investments, and several others. This funding follows a previous £10 million raised from Oxford Sciences Innovation during […]
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 hepatic porphyrias (AHPs), a group of rare genetic diseases. The trial marks a critical step forward in the development of targeted therapies for these debilitating conditions. The ENVISION trial has […]
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, Takeda’s fourth attempt to purchase Shire, comes after Shire had previously rejected three offers, citing undervaluation of its business and future growth prospects. Escalation in Acquisition Offers The latest bid […]
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 for SEK7.1 billion ($855 million). This strategic acquisition is set to significantly bolster Alexion’s offerings in the rare disease sector, particularly in treating Wilson disease, a rare copper-mediated disorder. Details […]
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 approximately $1.2 billion. This acquisition also involves the assumption of $360 million of Sucampo’s debt, signaling a significant investment by Mallinckrodt into expanding its specialty pharmaceutical and rare disease sectors. […]