EMA Grants BioMarin's Request For Speedy Review of Hemophilia A Gene Therapy

The European Medicines Agency (EMA) has granted its request for accelerated assessment review to BioMarin Pharmaceutical Inc’s BMRN valoctocogene roxaparvovec, gene therapy to treat severe hemophilia A, with an opinion expected in the first half of 2022, the company said.
With the EMA agreeing to give the therapy a closer look, BioMarin said it plans to submit an associated marketing application by June, based on Phase 3 data that includes one-year follow-up results and four and three years of follow-up from an ongoing Phase 1/2 dose-escalation study.
Meanwhile, BioMarin also expects to submit two-year follow-up safety and efficacy data from that Phase 3 trial to the FDA, with a potential resubmission of its application scheduled for Q2 2022.
The FDA shot down BioMarin’s application for the therapy back in August 2020, citing the need to see that two-year follow-up data.
In March, the FDA granted Regenerative Medicine Advanced Therapy designation to valoctocogene roxaparvovec.
Price Action: BMRN shares are up 0.09% at $78.17 during the market session on the last check Monday.