The New England Journal of Medicine (NEJM) has published phase 3 data from the XTEND-Kids study, which supports the potential of ALTUVIIIO to revolutionize treatment for children with severe hemophilia A, according to a statement from Sanofi - Aventis Groupe.
ALTUVIIIO (efanesoctocog alfa), a groundbreaking, high-sustained factor VIII replacement therapy, is authorized for use in both adults and children with hemophilia A. It is indicated for routine prophylaxis, on-demand treatment to control bleeding episodes, and perioperative management.
Sanofi highlighted that the pivotal XTEND-Kids study, detailed in the NEJM, demonstrated that ALTUVIIIO met both its primary and secondary endpoints. These included the absence of factor VIII inhibitor development and favorable annualized bleed rates (ABRs). Notably, no inhibitors to factor VIII were detected in patients treated with ALTUVIIIO. The study, which involved 73 patients, reported a median ABR of 0.00 and an estimated mean ABR of 0.61 (range: 0.42-0.90). In pediatric patients, the clearance rate of administered factor concentrates from the blood is typically higher than in adults. This often necessitates 2-4 injections per week when using standard half-life (SHL) or extended half-life (EHL) factor VIII products.
Moreover, Sanofi noted that 82% of children treated with once-weekly ALTUVIIIO experienced zero joint bleeds, suggesting that weekly prophylaxis with ALTUVIIIO could offer long-term joint health preservation.
ALTUVIIIO is currently approved and available in the United States, Taiwan, and Japan. On June 17, 2024, the European Commission approved ALTUVIIIO, marketed under the name ALTUVOCT, for the treatment and prevention of bleeds and perioperative prophylaxis in hemophilia A.
