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BioMarin's Hemophilia Gene Therapy Scores RMAT Designation In US


The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BioMarin Pharmaceutical Inc's (NASDAQ: BMRN) valoctocogene roxaparvovec investigational gene therapy for the treatment of adults with severe hemophilia A. 

  • RMAT is an expedited program intended to facilitate the development and review of regenerative medicine therapies.
  • In January, Phase 3 data were reported from patients with five bleeding episodes per year and using about 136-factor infusions per year.
  • Gene therapy treatment cut those average annualized numbers by 84% and 99%, respectively, to just under one and two.
  • In August last year, the Company received FDA Complete Response Letter for valoctocogene roxaparvovec.
  • The agency recommended that the Company complete the Phase 3 GENEr8-1 study and submit two-year follow-up safety and efficacy data.
  • In the EU, BioMarin is targeting submission of the marketing application with these results to the EMA in the second quarter of 2021, pending confirmation in planned pre-submission meetings.
  • Price Action: BMRN shares gained 0.66% at $78.22 in market trading hours on the last check Monday.

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Posted-In: gene therapy Hemophilia ABiotech News Health Care FDA General

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