BioMarin Pharmaceutical has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe haemophilia 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.
Recognising 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-commercialised 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.
The company remains on track to submit a Biologics License Application (BLA) to the US FDA by the end of the year. The FDA has granted valoctocogene roxaparvovec Breakthrough Therapy designation.
Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the EMA.