ALTUVIIIO received U.S. Food and Drug Administration (FDA) approval for the treatment of hemophilia A in adults and children prophylactically or on-demand to control bleeding episodes. The drug is a high-sustained factor VIII replacement therapy that is administered once weekly. ALTUVIIIO was previously granted fast track designation in February 2021 and orphan drug designation in August 2017.
Approval was based on the results of an open-label, non-randomized, interventional phase III study dubbed XTEND-1 and was granted under priority review. Results showed a 77% reduction in annual bleeding rates.
The treatment “delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis,” according to a press release from Sanofi.
“This approval marks an important clinical advancement for the hemophilia community because we have an option that can achieve higher levels of factor activity with a single simplified weekly dose,” said Lynn Malec, MD, medical director of Versiti Comprehensive Center for Bleeding Disorders, associate investigator at Versiti Blood Research Institute, and associate professor of medicine and pediatrics at the Medical College of Wisconsin. “By maintaining high levels of factor activity throughout the week, patients can be confident in the bleed protection ALTUVIIIO offers.”
ALTUVIIIO was also granted orphan drug designation in June 2019 by the European Commission, and regulatory submission is expected in the second half of 2023.
Source: Sanofi, February 23, 2023.