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BioMarin Unveils News Data From Late-Stage Hemophilia A Gene Therapy Candidate

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  • BioMarin Pharmaceutical Inc (NASDAQ: BMRN) has announced new data from GENEr8-1 Phase 3 study for valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.

  • Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.

  • Top-line one-year results from this study were previously communicated in January 2021.

  • New data presented include more details on annualized bleeding rate (ABR) in all study participants and annualized Factor VIII utilization rate.

  • Over 90% (N=134) of all participants in the GENEr8-1 study had an ABR of zero or a lower bleed rate than baseline after week four after the treatment.

  • Mean annualized Factor VIII utilization rate decreased from baseline on Factor VIII prophylaxis by 99% from 3961.2 to 56.9 after week four after treatment with valoctocogene roxaparvovec.

  • As previously shared in January 2021, a single dose of valoctocogene roxaparvovec significantly reduced mean ABR by 84% from a prospectively collected 4.8 baseline to 0.8 bleeding episodes per year.

  • Study participants also experienced a clinically meaningful increase in endogenous Factor VIII expression.

  • Last week, the European Medicines Agency validated BioMarin's resubmission of a marketing application for the gene therapy.

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