plasma-derived factor VIII + von Willebrand factor concentrate

Plasma-derived factor VIII + von Willebrand factor concentrate (pdFVIII/VWF) is a biologic/plasma-derived concentrate used for the treatment of hemophilia A, a rare X-linked bleeding disorder caused by deficiency of coagulation factor VIII (FVIII). The drug's active ingredients are plasma-derived coagulation factor VIII and von Willebrand factor (VWF), where VWF acts as a natural chaperone protein that masks critical FVIII epitopes and reduces immunogenicity. The biological targets are the coagulation cascade (specifically replacing deficient FVIII) and, through VWF, modulating immune responses to exogenous FVIII. This concentrate was the investigational treatment arm in the SIPPET trial (Survey of Inhibitors in Plasma-Product Exposed Toddlers), an independent, international, multicenter, prospective, randomized, open-label Phase 4 trial sponsored by Fondazione Centro Emofilia e Trombosi Angelo Bianchi Bonomi. The trial enrolled 251 previously untreated or minimally blood-component-treated male patients under age 6 with severe hemophilia A across 42 sites in 14 countries, comparing pdFVIII/VWF to recombinant FVIII with the primary objective of assessing inhibitor (neutralizing alloantibody) development during the first 50 exposure days or within 3 years of inclusion. The SIPPET results showed a significantly lower inhibitor incidence in the pdFVIII/VWF group compared to the recombinant FVIII group.

Brand names

Fanhdi

Other names

plasma-derived factor VIII/von Willebrand factor concentratepdFVIII/VWFhuman coagulation factor VIII / human von willebrand factorvon Willebrand factor/coagulation factor VIII complex (human)antihemophilic factor/von Willebrand factor complex (human)