BioMarin Pharmaceuticals has received full approval by the US Food and Drug Administration (FDA) for its gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for severe hemophilia A in adults. This marks the first gene therapy for patients with severe cases of the disease, which causes bleeding and progressive joint damage.
According to Emily Kimber, “BioMarin’s Roctavian, which is administered as a one-time infusion, delivers a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued haemophilia prophylaxis, relieving patients of their treatment burden.”
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(Source: PM Live, June 30th, 2023)