The U.S. Food and Drug Administration has approved Roctavian, an adeno-associated virus vector-based gene therapy for the treatment of adults with severe hemophilia A.
The FDA granted approval of Roctavian to BioMarin Pharmaceutical Inc.
The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. Roctavian was approved in Europe last year.
Hereditary hemophilia A is a potentially serious bleeding disorder. The rare genetic disease occurs due to a mutation on the gene which produces factor VIII (FVIII), a protein that enables blood to clot. Severe cases of hemophilia A can cause life-threatening health issues due to increased risk of uncontrolled bleeding.
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