BioMarin Plans Regulatory Submissions for Marketing Authorization of Valoctocogene Roxaparvovec to Treat Severe Hemophilia A in 4Q 2019 in both U.S. and Europe

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[{"type":"text","content":"SAN RAFAEL, Calif., July 8, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that based on recent meetings with health authorities in the U.S. and Europe, the company plans to submit marketing applications to both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 4Q 2019 for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. These submissions will be based on the updated three-year Phase 1/2 data and the recently completed Phase 3 interim analysis of patients treated with material from the to-be-commercialized process. Both submissions are expected to represent the first time a gene therapy product for any type of hemophilia will be reviewed for marketing authorization by health authorities.\n\n \nBoth the FDA and EMA continue to prioritize interactions related to the review of valoctocogene roxaparvovec through the Breakthrough Therapy Designation program in the U.S and the Priority Medicines (PRIME) regulatory initiative in Europe. Valoctocogene roxaparvovec is one of the first therapies to go through the new PRIME initiative. FDA and EMA grant expedited review designations for investigational therapies that address unmet medical needs in the treatment of a serious condition and/or show the potential to offer major therapeutic advantages over existing treatments. \n\"People with severe hemophilia A continue to experience clinically relevant breakthrough bleeds despite the current standard of care and can be limited in their physical activities,\" said Professor John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry; the chief investigator for the valoctocogene roxaparvovec Phase 1/2 study and a principal investigator for the Phase 3 study. \"Valoctocogene roxaparvovec represents a potentially transformative investigative therapy that could improve patients' quality of life, including consequences of bleeding, physical functioning, role functioning, emotional impact, treatment concern, and worry.\" \n\"We applaud the FDA's efforts to incorporate the patient voice in the regulatory review process. Powerful and moving testimonials from clinical study participants have helped serve as a critical element in the FDA's considerations of potentially the first commercially available gen...

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