The BLA was based on the P-III (XTEND-1) study evaluating efanesoctocog alfa (qw) in 159 patients aged ≥12yrs. with sev. hemophilia A prior treated with factor VIII replacement therapy. The US FDA’s decision is expected on Feb 28, 2023
The results showed clinical bleed protection & superiority to prior factor prophylaxis based on an intra-patient comparison & was well-tolerated, inhibitor development to factor VIII was not detected & provides high sustained factor activity levels in the normal to near-normal range
The company plans for regulatory submission of efanesoctocog alfa in the EU in 2023, based on (XTEND-Kids) pediatric study. The therapy has received BTD, ODD & FTD from the US FDA for hemophilia A
Ref: Globe Newswire | Image: Sanofi
