FDA Approves Sanofi’s Once-weekly ALTUVIIIO for Hemophilia A

The U.S. FDA has approved Sanofi’s ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management for adults and children with hemophilia A. 
 
ALTUVIIIO is the first and only hemophilia A treatment that 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.
 
Hemophilia A is a rare, lifelong condition in which the ability of a person’s blood to clot properly is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain.
 
This is the first approval of ALTUVIIIO. The FDA evaluated the application under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. The FDA previously granted ALTUVIIIO Breakthrough Therapy designation in May and Fast Track designation in February 2021, and Orphan Drug designation in August 2017.
 
Regulatory submission in the EU is anticipated in 2H23. The European Commission granted Orphan Drug designation in June 2019.
 
The approval is based on data from the pivotal XTEND-1 Phase 3 study demonstrating Once-weekly ALTUVIIIO prophylaxis met the primary endpoint, providing significant bleed protection for people with severe hemophilia A with a mean annualized bleeding rate (ABR) of 0.70 (95%) and a median ABR of 0. ALTUVIIIO met the key secondary endpoint with a significant reduction of 77% in ABR versus prior factor prophylaxis based on an intra-patient comparison (95% CI: 58%-78%).
 
Additional data showed prevention of joint bleeds with a median annualized joint bleeding rate of 0. Treatment with ALTUVIIIO provided 100% resolution of target joints, which are joints that have recurrent bleeds. ALTUVIIIO provided mean factor VIII activity greater than 40% for most of the week and greater than 10% at Day 7.