BioMarin's Roctavian becomes first gene therapy for hemophilia A approved by EC

25 August 2022
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US biotech BioMarin Pharmaceutical (Nasdaq: BMRN) yesterday announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to Roctavian (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).

The EC also endorsed the European Medicines Agency’s (EMA) recommendation for Roctavian to maintain orphan drug designation, thereby granting a 10-year period of market exclusivity. The EMA recommendation noted that, even in light of existing treatments, Roctavian may potentially offer a significant benefit to those affected with severe hemophilia A.

BioMarin’s shares edged up 1.4% yesterday and a further 1.3% to $94.15 in pre-market trading.

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