BioMarin: 4-Yr Data From Phase 3 Hemophilia Gene Therapy Study Shows Safety, Efficacy Of ROCTAVIAN

(RTTNews) - BioMarin Pharmaceutical (BMRN) announced new data supporting the long-term safety and efficacy of ROCTAVIAN. The company said the data from the longest Hemophilia Gene Therapy Study showed that durable and sustained bleed control and factor VIII expression maintained four years Post-ROCTAVIAN Infusion.

Hank Fuchs, president of Worldwide Research and Development at BioMarin, said: "Importantly, these Phase 3 data also indicate a plateauing of factor VIII levels after year three with the majority of patients remaining off prophylaxis, which shows ROCTAVIAN can offer long-term bleed protection for adults with severe hemophilia A and may provide relief from the burden of chronic infusions and injections."

ROCTAVIAN is an adeno-associated virus vector-based gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to adeno-associated virus serotype 5, which is determined by a blood test. The European Commission granted conditional marketing authorization to ROCTAVIAN on August 24, 2022. The FDA approved ROCTAVIAN on June 29, 2023.

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