BioMarin | pharmaphorum

Friday 5 December 2025

BioMarin

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Nektar, BioMarin, and Alnylam top list of biotech M&A target...

With Takeda’s £46 billion acquisition of Shire, it’s been a busy year for pharma M&A – and according to analysts Datamonitor, the specialist biotechs Nektar, BioMarin and Alnylam are th

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NICE rejects Biomarin's £520K per year rare disease drug

Company will negotiate access scheme

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‘Cure for hemophilia within reach’ as BioMarin and Spark gen...

While clinicians attending the American Society of Hematology meeting in Atlanta this weekend were anticipating a flurry of exciting data, the conference wasn’t entirely prepared for the ac

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Breakthrough status for BioMarin ratchets up haemophilia A competition

Breakthrough status for BioMarin ratchets up haemophilia A c...

The FDA has granted Breakthrough Therapy Designation to BioMarin’s gene therapy for haemophilia A, helping to intensify competition in the field.

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EU regulators back two more orphan drugs, question of pricin...

Two orphan medicines are heading towards the European market, after regulators recommended rare disease drugs from Biogen and BioMarin.

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New NICE rules could deny drugs to rare disease patients

Patients could be denied treatments, says BIA