European Medicines Agency Grants BioMarin's Request for Accelerated Assessment of Valoctocogene Roxaparvovec for Treatment of Severe Hemophilia A

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[{"type":"text","content":"MAA Resubmission on Track for June 2021\n\n\nSAN RAFAEL, Calif., May 24, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. Accelerated assessment reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP). A CHMP opinion is expected in the first half of 2022. \n\n \n \n \n \n \n \n\n \n\"BioMarin is pleased that the EMA has granted accelerated assessment for the review of valoctocogene roxaparvovec, which acknowledges its therapeutic innovative potential, and the unmet medical need that exists\" said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. \"We continue to work closely with the EMA to make valoctocogene roxaparvovec, the potential first gene therapy to treat hemophilia A, available as soon as possible.\"\nBioMarin plans to submit a Marketing Authorization Application (MAA) for valoctocogene roxaparvovec for the treatment of severe hemophilia A in June 2021. The submission will include 52 weeks of data from the Phase 3 GENEr8-1 study, as well as four and three years of follow-up from the respective dose cohorts in the ongoing Phase 1/2 dose escalation study. \nApplications are eligible for accelerated assessment if the CHMP and CAT decide the product is of major interest for public health, particularly from the point of view of therapeutic innovation. Evaluating a MAA under the EMA centralized procedure can take up to 210 days, not counting clock stops when applicants are requested to provide additional information. On request, the CHMP and CAT can reduce the time frame to 150 days if the applicant provides sufficient justification for an accelerated assessment, although an application initially designated for accelerated assessment can revert to the standard procedure during the review for a variety of reasons. The decision to grant accelerated assessment has no impact on the eventual CHMP and CAT opinion on whether a marketing authorization should be granted.\nRegulatory Status \nIn addition to today's update, in the United States, BioMarin intends to submit two-year f...

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