BioMarin Pharmaceutical (BMRN) has revealed new data that underline the long-term safety and efficacy of their gene therapy, ROCTAVIAN. The data stem from the longest-running study on hemophilia gene therapy to date, demonstrating sustained bleed control and stable factor VIII expression up to four years post-ROCTAVIAN infusion.
Hank Fuchs, president of Worldwide Research and Development at BioMarin, remarked, "Crucially, the Phase 3 data indicate factor VIII levels plateauing after the third year, with most patients no longer requiring prophylaxis. This underscores ROCTAVIAN's potential to provide enduring bleed protection for adults with severe hemophilia A, potentially alleviating the constant need for infusions and injections."
ROCTAVIAN employs an adeno-associated virus vector-based approach to treat adults with severe hemophilia A who lack antibodies against adeno-associated virus serotype 5, as confirmed through a blood test. The European Commission granted ROCTAVIAN conditional marketing authorization on August 24, 2022, and the FDA approved it on June 29, 2023.
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