(Boursier.com) — Data from the pivotal phase III XTEND-Kids study which evaluated ALTUVIIIO [facteur antihémophilique (recombinant), protéine de fusion Fc-VWF-XTEN]the first long-acting replacement factor VIII in its pharmacotherapeutic class, at a weekly prophylactic dose, in patients under 12 years of age with severe hemophilia A who have been treated before, have been presented, the June 25, as part of a clinical trial news session at the International Society on Thrombosis and Haemostasis (ISTH) Annual Meeting in Montreal, Canada.
The communication of Sanofi focused on the detailed results of the XTEND-Kids study devoted to ALTUVIIIO and confirmed that the primary endpoint of the study had been met – without giving rise to the development of anti-factor VIII inhibitors – as well as key secondary endpoints including annualized bleeding rate (ABR) and maintenance of factor VIII activity above pre-specified levels.
In children, the blood clearance of factor concentrates is higher than that observed in adults, which often makes it necessary to inject them with conventional or extended half-life replacement factors VIII 2 to 4 times a week. These data confirm that a weekly dose of 50 IU/kg of ALTUVIIIO provides highly effective protection against bleeding in both adults and children and that this regimen is suitable for all clinical scenarios..
ALTUVIIIO is the first long-acting and sustained-acting factor VIII replacement in its pharmacotherapeutic class. It was approved in February 2023 by the United States Food and Drug Administration (FDA) for routine prophylaxis and on-the-spot treatment to control bleeding episodes, as well as for perioperative (surgical) management, in adult and child with hemophilia A. It is the first replacement factor VIII to receive Breakthrough Therapy designation from the FDA, which it obtained in May 2022. The FDA awarded it also granted an accelerated evaluation (Fast Track) in February 2021, as well as orphan medicinal product designation in 2017. The European Commission granted it orphan medicinal product designation in June 2019 and the European Medicines Agency granted it an authorization on the market in May 2023.
©2023 Boursier.com