Source – Sanofi
On June 26, 2023, at the Annual Meeting of the International Society on Thrombosis and Haemostasis (ISTH) in Montreal, Canada, significant findings were unveiled from the Phase III XTEND-Kids study examining the efficacy of Altuviiio (Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein) as a once-weekly prophylactic treatment for severe hemophilia A in children under 12 years of age. This breakthrough therapy, a high-sustained factor VIII replacement therapy, showcased remarkable results and met its primary endpoint by demonstrating no inhibitor development to factor VIII. The study also confirmed positive secondary endpoints, including maintaining factor VIII activity above specified levels and reducing the annualized bleeding rate (ABR).
Typically, pediatric patients require more frequent injections of factor concentrates due to faster clearance in their bloodstream compared to adults. Standard (SHL) or extended half-life (EHL) factor VIII products often necessitate 2-4 weekly injections. However, the data presented affirmed that a once-weekly dose of Altuviiio at 50 IU/kg provides highly effective bleed protection for both children and adults, proving its versatility in various clinical scenarios.
Hemophilia A is a rare lifelong condition characterized by impaired blood clotting, resulting in excessive bleeding and spontaneous bleeds into joints that can lead to chronic pain, joint damage, and diminished quality of life. The severity of hemophilia is determined by the level of clotting factor activity, with lower activity levels correlating with an increased risk of bleeding.
Altuviiio, a pioneering high-sustained factor VIII replacement therapy, gained approval from the US Food and Drug Administration (FDA) in February 2023 for routine prophylaxis, on-demand treatment, and perioperative management (surgery) in both adults and children with hemophilia A. It was granted Breakthrough Therapy designation by the FDA in May 2022, making it the first factor VIII therapy to receive this recognition. Altuviiio also received Fast Track designation in February 2021 and Orphan Drug designation in 2017. The European Commission granted Orphan Drug designation in June 2019, and the European Medicines Agency accepted the Marketing Authorization Application (MAA) for efmoroctocog alfa in May 2023.
“The results from XTEND-Kids mark an important breakthrough as we strive for optimized bleed protection as the standard of care. Achieving high-sustained factor activity with once weekly dosing means a freedom from the tradeoffs between treatment burden and efficacy we often see in treating severe hemophilia A. The reliable and consistent bleed protection Altuviiio provides offers confidence for children living with hemophilia and their families to manage hemophilia with less worry.”
– Lynn Malec, MD, Associate Professor of Medicine and Pediatrics at The Medical College of Wisconsin
