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.
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- 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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BMRNBioMarin Pharmaceutical Inc
$60.223.42%
Edge Rankings
Momentum
16.80
Growth
N/A
Quality
28.59
Value
65.43
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