BioMarin’s Roctavian Gets EC Conditional Marketing Authorization for Severe Hemophilia A

BioMarin Pharmaceutical’s gene therapy Roctavian (valoctocogene roxaparvovec) has received a conditional marketing authorization from the European Commission (EC) for treatment of patients with severe hemophilia A.

The one-time infusion treatment is the first approved gene therapy for hemophilia A, a condition that includes a mutation in the gene responsible for producing Factor VIII, a protein needed for blood clotting.

The treatment delivers a functional gene designed to enable the body to produce Factor VIII on its own.

The therapy is specifically authorized for adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5.