Shots:

• The two-year analysis of the P-III (GENEr8-1) study evaluates the superiority of valoctocogene roxaparvovec (6e13 vg/kg) vs FVIII prophylactic therapy in 134 patients with sev. Hemophilia A
• The results showed a reduction in ABR, 85% from a baseline in the pre-specified primary analysis in patients from a prior non-interventional study with a median follow-up of 110wks., improved bleed free rate from 32% at baseline maintained through Year 1 (82%) & Year 2 (84%), 95% of patients remain off Factor VIII prophylactic therapy
• Additionally, no patients developed inhibitors to Factor VIII, malignancy, or thromboembolic events, no new safety signals were observed with no treatment-related SAE

to­Ref: BioMarin | Image: UCLB