Phase 3 study of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A

Robert Sidonio, Jr., M.D., M.Sc.
Hemophilia of Georgia Center for Bleeding and Clotting Disorders of CHOA
Atlanta, GA, U.S.

Inhibitor development remains a serious complication of factor VIII replacement therapy in previously untreated patients (PUPs) with hemophilia A, with reported incidence rates around 30%. In this presentation, Robert Sidonio shares findings from a phase 3, open-label, multicenter study evaluating rurioctocog alfa pegol (rAHF-PEG), an extended half-life recombinant FVIII, in PUPs. Seven patients underwent immune tolerance induction with rAHF-PEG, which was successful in five and partially successful in one. Annualized bleeding rates were lower in patients receiving prophylaxis compared to on-demand treatment. Most bleeds resolved with one to two infusions, with excellent or good hemostatic efficacy. These results support the safety and efficacy of rAHF-PEG in this population, including its potential role in immune tolerance induction.