Biomarin presents new five-year data for hemophilia gene therapy

Gene therapy valoctocogene roxaparvovec had a turbulent start, but Biomarin has now released five-year data for the treatment, which is crucial to getting FDA approval.

Photo: Helle Arensbak/IND

While Biomarin waits for the US Food and Drug Administration (FDA) to make a decision on the company's gene therapy in hemophilia A, Biomarin publishes five-year highlights of clinical data from studies with the drug.

The FDA specifically requested five-year data as one of its requirements last year, when it rejected Biomarin's first application.

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