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BioMarin : Bleed Control In Phase 3 Study Of Valoctocogene Roxaparvovec On Severe Hemophilia A

BioMarin Pharmaceutical Inc. (BMRN) announced stable and durable annualized bleed control in phase 3 GENEr8-1 study of valoctocogene roxaparvovec in adults with Severe Hemophilia A. The 134-participant study met all primary and secondary efficacy endpoints at two year analysis.

In the study, Annualized Bleeding Rate was significantly reduced by 4.1 treated bleeds per year or 85% from a baseline mean of 4.8 in the pre-specified primary analysis in participants from a prior non-interventional study.

Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII infusion rate in the rollover population by 133 infusions per year or 98% from baseline.

At the end of the second year post-infusion with valoctocogene roxaparvovec, participants in the modified intent-to-treat population had a mean endogenous Factor VIII activity level of 23.0 U/dL, as measured by the chromogenic substrate assay and 36.1 IU/dL, as measured by the one-stage assay.

The European Medicines Agency validated BioMarin's resubmission of a Marketing Authorization Application and a Committee for Medicinal Products for Human Use and Committee for Advanced Therapies opinion is anticipated in the first half of 2022.

BioMarin said it is planning to meet with the U.S. Food and Drug Administration to discuss the resubmission of a Biologics License Application targeted for the second quarter of 2022, followed by an expected six-month review by the FDA.

Valoctocogene roxaparvovec has received both Regenerative Medicine Advanced Therapy (RMAT) designation and Breakthrough Therapy Designation from FDA, which are intended to expedite development of drugs for serious or life-threatening diseases and conditions.

In addition to the RMAT Designation and Breakthrough Therapy Designation, valoctocogene roxaparvovec also has received Orphan Drug Designation from the FDA and EMA for the treatment of severe hemophilia A.

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries.

Additionally, people with the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints.

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