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BioMarin submits gene therapy for US FDA review; filing accepted by EMA

BioMarin Pharmaceutical Inc. submitted a biologics license application for its gene therapy valoctocogene roxaparvovec to the U.S. Food and Drug Administration and received acceptance of the treatment's application from the European Medicines Agency.

The San Rafael, Calif.-based biotechnology company's therapy treats adult patients with hemophilia A, a disorder that causes potentially life-threatening bleeding complications resulting from a missing or defective factor VIII protein.

The company's submissions of valoctocogene roxaparvovec are based on an ongoing phase 1/2 trial in which the drug reduced bleeding in hemophilia patients by 96% over three years and internal data from an ongoing phase 3 trial.

BioMarin expects the review processes for the drug to start in January 2020 for the EMA and February 2020 for the FDA.

In October, the EMA granted valoctocogene roxaparvovec accelerated assessment, which reduces review time to 150 days from 210 days, although the agency's support for the drug dates back to 2017 when it gave the drug access to its priority medicines regulatory initiative.

Both the EMA and the FDA have given the therapy orphan designations and the U.S. regulator has also tagged it as a breakthrough therapy.