BioMarin's hemophilia gene therapy Roctavian wins conditional EU backing amid FDA delay

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via The European Medicines Agency has recommended granting a conditional approval to BioMarin’s Roctavian, also known as valoctocogene roxaparvovec, to treat patients with severe hemophilia A who do not have factor VIII inhibitors, the European regulator said Friday. Patients should also test negative for antibodies to the adeno-associated virus serotype 5, which serves as the vector for the gene therapy.

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