BioMarin down big on FDA rejection of gene therapy for hemophilia A

Aug. 19, 2020 8:34 AM ETBioMarin Pharmaceutical Inc. (BMRN)BMRNBy: Douglas W. House, SA News Editor23 Comments
  • 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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