Efanesoctocog alfa

Efanesoctocog alfa (BIVV001) is a recombinant factor VIII (FVIII) analogue fusion protein used for the routine prophylaxis, perioperative management of bleeding and on-demand treatment and control of bleeding episodes in patients with hemophilia A. The use of FVIII replacement products is beneficial in patients with hemophilia A; however, their quality of life can be affected due to frequent doses. Efanesoctocog alfa was designed to have an extended half-life, and to surpass the half-life ceiling to which other forms of recombinant FVIII are subjected due to their association with von Willebrand factor (VWF). Endogenous VWF protects FVIII from degradation but also sets a half-life of approximately 15 to 19 h. To extend the half-life of FVIII, efanesoctocog alfa is fused to dimeric Fc, a D'D3 domain of VWF, and two XTEN polypeptides.

In February 2023, efanesoctocog alfa was approved by the FDA as a new class of factor VIII therapy for hemophilia A.

Efanesoctocog alfa is indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes, and perioperative management of bleeding.