BioMarin’s Roctavian (valoctocogene roxaparvovec) Receives EC’s Conditional Marketing Authorization for Severe Hemophilia A


The EC has granted CMA to Roctavian for sev. hemophilia A in adult patients without a history of factor VIII inhibitors & without detectable Ab to AAV5
The EC’s decision was based on the clinical development program incl. 2yr. results from the P-III (GENEr8-1) study showed a stable & durable bleed control with a reduction in mean ABR & mean annualized factor VIII infusion rate
In the ongoing P-I/II dose escalation study, the results included 5 & 4yr. of follow-up from the 6e13 & 4e13vg/kg dose groups while 6e13vg/kg dose was well tolerated with no delayed-onset TRAEs. Roctavian continues to be made available to eligible patients with sev. haemophilia A & the company is expected to resubmit the BLA at the end of Sept. 2022

Ref: BioMarin | Image: BioMarin