FDA Gives BioMarin Pharmaceuticals' Hemophilia Gene Therapy Priority Review
The Food and Drug Administration (FDA) has given BioMarin Pharmaceutical (NASDAQ: BMRN) a priority review for its valoctocogene roxaparvovec (valrox) gene therapy to treat patients with hemophilia A. The priority review will shave four months off of the review time for the marketing application, compared to a standard review. The agency expects to make a decision by Aug. 21, 2020.
If approved, valrox would be the first gene therapy approved to treat hemophilia A. Currently, patients are treated with a protein called Factor VIII, which has to be infused two to three times per week and still doesn't prevent patients from all bleeding events. Valrox restores expression of Factor VIII in a patient's cells, eliminating the need for the replacement therapy.
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Source Fool.com
