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EMA grants PRIME access to BioMarin's hemophilia A therapy

The European Medicines Agency granted access to the priority medicines regulatory initiative to BioMarin Pharmaceutical Inc.'s investigational gene therapy drug for severe hemophilia A, which causes increased bleeding and usually affects males.

The agency's Committee for Medicinal Products for Human Use determined the PRIME access was justified due to the therapy's preliminary clinical data in affected patients, supporting that a single IV administration results in sustained restoration of factor VIII activity, reduction of annualized bleeding rates and improved quality of life.