Efanesoctocog Alfa: First Approval.

Efanesoctocog alfa (ALTUVIIIOTM; [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl]), a von Willebrand factor (VWF) independent, recombinant DNA-derived Factor VIII (FVIII) concentrate, has been developed by Bioverativ Therapeutics, Inc (a Sanofi company) and Swedish Orphan Biovitrum AB (Sobi). Efanesoctocog alfa was approved in February 2023 in the USA for use in adults and children with hemophilia A (congenital FVIII deficiency) for: routine prophylaxis to reduce the frequency of bleeding episodes; on-demand treatment and control of bleeding episodes; perioperative management of bleeding. This article summarizes the milestones in the development of efanesoctocog alfa leading to this first approval for hemophilia A.