BioMarin Pharmaceutical (NASDAQ:BMRN) slumps 22% premarket on increased volume in reaction to its announcement that it received a Complete Response Letter (CRL) from the FDA regarding its marketing application seeking approval of gene therapy valoctogene roxaparvovec for the treatment of severe hemophilia A.
The CRL cited the need for additional data (two years) from the ongoing Phase 3 study, 270-301, to confirm the duration of the treatment effect based on the primary endpoint of annualized bleeding rate. The review team recommended that the company complete the study and submit the two-year results. As such, the timeline for the data filing will be late 2021/early 2022 since the last participant will complete the follow-up in November 2021.
The FDA’s action date was August 21.
It plans to meet with the agency in the coming weeks to clarify next steps.
Its marketing application in Europe is currently under EMA review.
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