Efanesoctocog alpha is a high sustained Factor VIII replacement therapy designed to decouple recombinant Factor VIII from endigenous von Willebrand factor and overcome the von Willebrand factor imposed half-life ceiling.
It is a recombinant factorVIII molecule bioengined to circulate independently of endigenous von Willebrand factor.
Factor VIII has high affinity interaction with von Willebrand vector, which protects factor VIII from premature breakdown and rapid clearance.
Efanesoctocog alpha in severe hemophilia A provided once weekly had superior bleeding prevention to pre-study prophylaxis, normal to near normal factor VIII activity and improvements in physical health, pain, and joint health.
Efanesoctocog alpha is composed of a single recombinant factor VIII protein and three additional components that contribute to its increased half-life.
It consists of an Fc-infused B domain deleted factor VIII protein covalently linked to an exaggeration fragment of VWF, preventing binding to endogenous VWF.
As compared to factor VIII showed a half-life that was four times longer and an area under the curve that was six times longer.
In children with severe hemophilia prophylactic therapy once a week with Efanesoctocog alpha provided sustained Factor VIII activity to the normal or near normal range of greater than 40 international units per deciliter for three days and more than 10 units per deciliter for almost 7 days after it administration: it led to effective bleeding prevention EXTEND-Kids trial group).