BioMarin Reports Results of Valoctocogene Roxaparvovec in P-III GENEr8-1 Study for Hemophilia A

Shots:

• The ongoing P-III GENEr8-1 study involves assessing of valoctocogene roxaparvovec (6e13 vg/kg dose) in 134 patients with with severe hemophilia A. The patients received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up
• The study met 1EPs & 2EPs i.e. reduction in ABR by 84% demonstrating superiority to factor VIII prophylaxis and reduced mean annualized factor VIII infusion rate by 99%. In subset dosed >2yrs. ago, rate of decline in factor VIII expression is slow
• Mean ABR in the population was 0.9 over these two-plus yrs. BioMarin plans to meet with EMA to discuss the submission of 1yrs. data and the FDA to review 2yrs. data-request

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