Recombinant coagulation factor VIII Fc-von Willebrand factor fusion protein Treatment of hemophilia A

Congenital hemophilia A and B care has improved drasti-cally in recent years. Prophylaxis in severe hemophilia pa-tients allows them to have low annual bleeding rates, less arthropathy and better quality of life. Although prophylaxis has improved outcomes in these patients, there still are some unmet needs regarding the number of infusions per week, maintaining higher trough levels and reducing treatment burden. Extended half-life factor VIII (FVIII) products are now available, but with a modest improvement in dose frequency. Considering that FVIII circulates in a complex with von Will-ebrand factor (VWF), which stabilizes and protects FVIII from degradation and subjects FVIII to a half-life of 15-19 h, a new rFVIII decoupled from endogenous VWF has been developed. Efanesoctocog alfa (BIVV-001) is a recombinant FVIII Fc fusion protein molecule attached with the FVIII binding domain of VWF, D ' D3, and 2 XTEN linkers. Phase I/II trial results present no safety concerns, high and sustained FVIII activity levels, and a half-life almost 4 times greater than those of other rFVIII products. Clinical trials are still running, but early re -sults show a safe and effective treatment option for patients with hemophilia.