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€1.5m Price Tag Could Spell EU Success For BioMarin’s Hemophilia Gene Therapy

First Hemophilia A Gene Therapy Approval

 
• By Andrew McConaghie

The approval is a breakthrough for BioMarin, which is hoping to seize market share from Factor VIII replacement therapies and Roche’s Hemlibra.

Brisbane, CA, USA - Mar 1, 2020: The BioMarin logo seen at American biotechnology company BioMarin Pharmaceutical Inc.'s office in Brisbane, California.
The EU is vital for Roctavian's prospects, and the wider European and Middle East market two and half times larger than the US. • Source: Shutterstock

BioMarin’s persistence with Roctavian (valoctocogene roxaparvovec) has paid off, with the European Commission making it the first hemophilia A gene therapy approved anywhere in the world.

The news is not a surprise as the EU’s medicines regulator had recommended approval in June, but is nevertheless a triumph for the company, which saw the drug rejected by the US Food and Drug

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