Sanofi Global Health announced that the US Food and Drug Administration (FDA) has granted priority review to the Biologics License Application (BLA) for efanesoctocog alfa, a novel investigational recombinant factor VIII replacement therapy for people with hemophilia A. The review, announced by news release, has an FDA target decision date of February 28, 2023.
“Factor therapy remains a cornerstone of hemophilia treatment, but innovation has been needed in this area to address challenges related to bleed protection and cumbersome treatment regimens,” Steve Pipe, MD, professor and director of the Pediatric Hemophilia and Coagulation Disorders Program at the University of Michigan in Ann Arbor, wrote.
“If approved, efanesoctocog alfa can deliver close to normal factor activity levels for the majority of the week, potentially offering a new tier of protection. Such therapeutic benefits would represent important advances in unmet medical needs for people with hemophilia A and may transform the prophylactic treatment landscape.”
The results of the XTEND-1 phase 3 study support the BLA by demonstrating clinically meaningful prevention of bleeding and greater efficacy in terms of reduced annualized bleeding rates compared to previous factor prophylaxis, as well as good tolerability and no detected inhibitor development in the participants.
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The open-label, nonrandomized study was conducted on 159 people with severe hemophilia A, and the results were presented at the 30th International Society of Thrombosis and Haemostasis Congress. The main treatment-related adverse events of efanesoctocog alfa were headache, falls, arthralgia, and back pain.
Efanesoctocog alfa is an investigational recombinant factor VIII prophylactic treatment designed to be administered once weekly to prevent bleeds. It is the first factor VIII therapy that overcomes the von Willebrand factor ceiling, which significantly limits the efficacy of existing factor VIII therapies.
FDA grants priority review to efanesoctocog alfa for people with hemophilia A. News release. Sanofi; August 30, 2022.