The European Commission has granted conditional marketing authorization to valoctocogene roxaparvovec (Roctavian™), a novel gene therapy for patients with severe hemophilia A, BioMarin Pharmaceutical Inc. announced.

“This approval in the [European Union] represents a medical breakthrough in the treatment of patients with severe hemophilia A that expands the conversation between a patient and physician on treatment choices to now include a one-time infusion that protects from bleeds for several years,” professor Johannes Oldenburg, director of the Institute of Experimental Haematology and Transfusion Medicine and the Haemophilia Centre at the University Clinic in Bonn, Germany, said.

BioMarin plans to resubmit a Biologics License Application (BLA) to the US Food and Drug Administration for valoctocogene roxaparvovec by the end of September 2022. They anticipate its review could take up to 9 months.

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The conditional marketing authorization in Europe is based on positive results from the clinical development program of valoctocogene roxaparvovec, which included outcomes from the phase 3 global GENEr8-1 study. This 2-year study showed durable reduced mean annualized bleeding rates (ABR) and annualized factor VIII infusion rates among patients.

Additional studies on valoctocogene roxaparvovec include a phase 3B study assessing the safety and efficacy of the 6e13 vg/kg dose with prophylactic corticosteroids in patients with hemophilia A and 2 phase 1/2 studies exploring valoctocogene roxaparvovec in patients with hemophilia A who had adeno-associated virus type 5 (AAV5) antibodies or factor VIII inhibitors.

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No delayed-onset treatment-related adverse events have been recorded among patients administered a single 6e13 vg/kg dose of valoctocogene roxaparvovec. Common early adverse events included mild elevations in liver enzymes, transient infusion-related reactions, nausea, headache, and fatigue. No patients developed factor VIII inhibitors, thromboembolic events, or malignancies in association with the treatment.

Conversion from the conditional marketing authorization to a standard authorization will depend on the long-term results of the GENEr8-1 study and the study of valoctocogene roxaparvovec in combination with prophylactic corticosteroids.


First gene therapy for adults with severe hemophilia A, BioMarin’s Roctavian™ (valoctocogene roxaparvovec), approved by European Commission (EC). News release. BioMarin Pharmaceutical Inc.; August 24, 2022.