Shots:

• The P-I/II study evaluates valoctocogene roxaparvovec in adults with sev. hemophilia A. Additionally, 6e13vg/kg & 4e13 vg/kg cohorts were followed for 6 & 5yrs. after treatment & valoctocogene roxaparvovec exhibited maintained hemostatic effectiveness
• At the time of the data cut, patients in the 6e13/4e13 vg/kg cohort remain off prophylactic Factor VIII treatment, mean cumulative ABR remains 1≤ & 0.7, 95% & 91% reduction in mean ABR, 96% & 93% reduction in mean annualized factor VIII usage from baseline @6 & 5yrs.
• The company is expected to resubmit a BLA for valoctocogene roxaparvovec to the US FDA at the end of Sept. The MAA is under EMA’s review with an expected CHMP opinion in mid-2022

Ref:  BioMarin | Image: BioMarin