The European Medicines Agency has validated BioMarin’s application to market its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.
As such, the company said it expects the agency’s review of the therapy in January next year under accelerated assessment.
The EMA granted access to its Priority Medicines (PRIME) regulatory initiative in 2017 for valoctocogene roxaparvovec and recently granted BioMarin’s request for accelerated assessment of the MAA, potentially shortening the review period.
The submission is based on an interim analysis of study participants treated in an ongoing Phase III study with material from the to-be-commercialised process and updated three-year Phase I/II data.
It marks the first marketing application to be filed in Europe for a gene therapy product for any type of hemophilia.
BioMarin also announced the filing of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the treatment, with the review expected to being in February.
“We are pleased that the agency has recognised the potential scientific advancement that valoctocogene roxaparvovec could bring to people with severe hemophilia A,” said Hank Fuchs, 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.”
