Biomarin’s hemophilia gene therapy nears European market after US setback
A single dose of the US-based biotech firm’s drug could be enough for a lifelong treatment of the rare bleeding disorder – but Roctavian’s price tag is making governments around the world think twice.
By James Paton, bloomberg
Biomarin Pharmaceutical Inc. expects European patients to begin receiving its gene therapy for a life-threatening blood-clotting disorder before the end of the year, raising the drugmaker’s hopes it can succeed where rival Bluebird Bio Inc. faltered.
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