BioMarin Pharma hassubmitted a MAA to the European Medicines Agency for valoctocogene roxaparvovec for adults with severe hemophilia A.

BioMarin Pharmaceutical Inc. has announced that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec,(Valrox) 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.

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 III study with material from the to-be-commercialized process and updated three-year Phase 1/II data. This submission marks the first marketing application submission 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 .