Nov 21,2019: BioMarin Pharmaceutical Inc. announced 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.Â
BioMarin is going to provide an update in January 2020. This submission will results in the first marketing application submission for a gene therapy product for any type of hemophilia.
If satisfactory justification for an accelerated assessment provided by the applicant time-frame will reduced to 150 days byThe CHMP and CAT, although an application initially designated can revert to the standard procedure during the review for a multiple reasons.
https://investors.biomarin.com/2019-11-21-BioMarin-Submits-Marketing-Authorization-Application-to-European-Medicines-Agency-for-Valoctocogene-Roxaparvovec-to-Treat-Severe-Hemophilia-A
