BioMarin Pharmaceutical (BMRN -0.3%) discloses that the European Medicines Agency (EMA) has requested complete 52-week data from the 134 participants in its ongoing Phase 3 clinical trial, BMN 270-301, evaluating gene therapy valoctocogene roxaparvovec in patients with severe hemophilia A.
The last follow-up visit should happen in November which will enable the company to submit the data by the end of Q1 2021. As a result, the EMA’s accelerated review of the marketing application will revert to standard review.
The company encountered a similar issue in the U.S. Last month, it received a CRL from the FDA citing the need for two-year data from the trial to confirm the duration of the treatment effect. The timeline for submitting the requested results will be late 2021/early 2022 since the last follow-up visit will not be completed until November of next year. The agency’s previous action date was August 21.
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