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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.
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