BioMarin Pharma Announces FDA Approval Of ROCTAVIAN Gene Therapy

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) announced the FDA approved ROCTAVIAN gene therapy for the treatment of adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. The company noted that the one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S.

The company said the FDA approval is based on data from the global Phase 3 GENEr8-1 study, the largest Phase 3 trial of any gene therapy in hemophilia. BioMarin will conduct an extension study that will follow all clinical trial participants for up to 15 years, and post-approval studies to follow those dosed in a real-world setting for 15 years or more.

The European Commission granted conditional marketing authorization to ROCTAVIAN on August 24, 2022.

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