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BioMarin Issues Additional Data From Valoctocogene Roxaparvovec Study

BioMarin Pharmaceutical Inc. (BMRN) announced additional data from its four-year update of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for severe hemophilia A. The data is the most current data (April 8, 2020, cut off) and includes four years of data for the 6e13 vg/kg cohort and three years of data for the 4e13 vg/kg cohort.

Hank Fuchs, President, Global Research and Development at BioMarin, said: "Demonstrating a 96% reduction in exogenous Factor VIII usage as patients are now producing their own endogenous factor VIII is a potential benefit that we hope to be able to offer as we work closely with regulators to seek approval and work to reduce the burden of hemophilia."

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