Five-Year Data Of BioMarin's Hemophilia Gene Therapy Shows Sustained Benefit

  • 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.
  • Related content: Benzinga's Full FDA Calendar

Posted In: Briefsgene therapyHemophilia APhase 2 TrialBiotechNewsHealth CareFDAGeneral

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