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 (NASDAQ: 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.

See more from Benzinga

• Click here for options trades from Benzinga

• Five-Year Data Of BioMarin's Hemophilia Gene Therapy Shows Sustained Benefit

© 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.