BioMarin Submits Marketing Authorization Application to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A

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[{"type":"text","content":"SAN RAFAEL, Calif., Nov. 21, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. Subject to completion of EMA's validation check, BioMarin anticipates the start of the MAA review to commence in January 2020 under accelerated assessment. BioMarin will provide an update in January 2020. \n\n \nRecognizing valoctocogene roxaparvovec for its potential to benefit patients with unmet medical needs, EMA granted access to its Priority Medicines (PRIME) regulatory initiative in 2017 and recently granted BioMarin's request for accelerated assessment of this MAA, potentially shortening the review period. This submission is based on an interim analysis of study participants treated in an ongoing Phase 3 study with material from the to-be-commercialized process and updated three-year Phase 1/2 data. This submission marks the first marketing application submission for a gene therapy product for any type of hemophilia. \nAccelerated 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). Applications 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.\nThe company remains on track to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the end of the year. The FDA has granted v...

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