BioMarin Pharma (BMRN) Announces EMA Validates its MAA for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A

December 23, 2019 8:32 AM EST

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BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January 2020 under accelerated assessment.

Recognizing 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 in Europe for a gene therapy product for any type of hemophilia.

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). 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.

The U.S. Food and Drug Administration (FDA) has granted valoctocogene roxaparvovec Breakthrough Therapy designation. Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the EMA.

"We are pleased that the agency has recognized the potential scientific advancement that valoctocogene roxaparvovec could bring to people with severe hemophilia A," said Hank Fuchs, M.D., President, Global Research and Development at BioMarin. "We continue to move thoughtfully and urgently through the regulatory review process to deliver a treatment that we believe has the potential to make a meaningful difference to people with hemophilia A. We also believe that the field of gene therapy offers the possibility of changing the practice of medicine in many rare genetic diseases like hemophilia A."



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