Five-Year Data Of BioMarin's Hemophilia Gene Therapy Shows Sustained Benefit - Biomarin Pharmaceutical (NASDAQ:BMRN)

BioMarin Pharmaceutical Inc BMRN has announced an update to its previously reported results from Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A.
Data will be shared at the International Society on Thrombosis and Haemostasis 2021 Virtual Congress to be held in July.
Five-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts show a sustained treatment benefit of gene therapy.
All participants in both cohorts remain off prophylactic Factor VIII treatment.
The mean annualized bleed rates (ABR) in year five for the 6e13 vg/kg cohort was 0.7 with an ABR reduction of 95% and Factor VIII use reduction of 96% through five years, compared to pre-infusion.
The mean ABR in year four for the 4e13 vg/kg cohort was 1.7, with a mean cumulative ABR reduction of 92% and Factor VIII use reduction of 95% through four years, compared to pre-infusion.
The overall safety profile of valoctocogene roxaparvovec in the Phase 1/2 study remains consistent with previously reported data with no delayed-onset treatment-related adverse events.
In Europe, BioMarin plans to submit a marketing application in June, based on one-year results from the Phase 3 study.
In the U.S., the company plans to submit two-year follow-up safety and efficacy data from the Phase 3 study.
BioMarin is targeting a Biologics License Application submission in the second quarter of 2022.
Price Action: BMRN shares are down 1.92% at $76.18 on the last check Wednesday.
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