Business
BioMarin's Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA with Review Action Date of August 21, 2020
No Advisory Committee Meeting Currently Planned to Review the Application If approved, 1st Gene Therapy in U.S. for the Treatment of Any Type of Hemophilia
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"No Advisory Committee Meeting Currently Planned to Review the Application\n If approved, 1st Gene Therapy in U.S. for the Treatment of Any Type of Hemophilia\n FDA Also Accepts Premarket Approval (PMA) Application for Companion Diagnostic Test, a 1st for a Gene Therapy\n\n\nSAN RAFAEL, Calif., Feb. 20, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A. This acceptance by the FDA marks the first marketing application accepted for a gene therapy product for any type of hemophilia in the United States. \n\n \n \n\n \nThe FDA has granted priority review designation to valoctocogene roxaparvovec, which is granted to drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. The Prescription Drug User Fee Act (PDUFA) action date is August 21, 2020. The FDA has informed the company that they are not currently planning to hold an advisory committee meeting to discuss the application. \nThe application is based on a Phase 3 interim analysis of study participants treated with investigational product manufactured by the to-be-commercialized process and three-year Phase 1/2 data. BioMarin has constructed, commissioned, and validated one of the first gene therapy manufacturing facilities of its kind in the world. This award-winning facility is located in Novato, California. Marketing authorization documentation has been included in the applications, and the facility is ready for inspection to support approval. \nIn addition, the FDA has accepted the premarket approval (PMA) application for an AAV5 total antibody assay intended as a companion diagnostic test for valoctocogene roxaparvovec. With a low prevalence of pre-existing immunity to AAV5, BioMarin estimates that approximately 80% of people with hemophilia A in the US do not have preexisting immunity to AAV5 that would make them ineligible for AAV5-mediated gene therapy treatment. The assay is produced by ARUP Laboratories, a leading national reference laboratory and a nonprofit enterprise of the Univ...