BMRN - BioMarin granted FDA nod for hemophilia A gene therapy

2023-06-29 15:00:48 ET

• BioMarin Pharmaceutical ( NASDAQ: BMRN ) announced Thursday that the FDA approved its one-time gene therapy, Roctavian, as a treatment for adults with the bleeding disorder severe hemophilia A.
• The approval was based on data from a global Phase 3 study in which 112 patients who received Roctavian indicated a 52% reduction in annualized bleeding rates (ABR) compared to their baseline ABR after the end of a three-year follow-up.
• According to the company, Roctavian is the first approved gene therapy for severe hemophilia A in the U.S. To be eligible to receive Roctavian, patients should not test positive for antibodies to adeno-associated virus serotype 5 (AAV5) in an FDA-approved test.
• The European Medicines Agency cleared Roctavian for hemophilia A in August 2022, and in April, the company lowered the full-year guidance for the therapy to $50M – $150M citing a delay in the FDA’s review of its marketing application.

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