The FDA granted priority review for the Bioloigics License Application (BLA) Sanofi submitted for efanesoctocog alfa indicated for individuals with hemophilia A with a decision date set for February 28, 2023.
The US Food and Drug Administration (FDA) granted priority review for the Bioloigics License Application (BLA) Sanofi submitted for efanesoctocog alfa indicated for individuals with hemophilia A. The decision is expected February 28, 2023.
Sanofi developed the novel investigational recombinant factor VIII therapy to extend protection from bleeds in individuals living with the life-threatening rare condition. This treatment would require a single weekly prophylactic dosing.
“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. 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," Steve Pipe, MD, Professor and Director of Pediatric Hemophilia and Coagulation Disorders Program, University of Michigan, said in a statement.
"Such therapeutic benefits would represent important advances in unmet medical needs for people with hemophilia A and may transform the prophylactic treatment landscape,” he continued.
Data from the phase 3 XTEND-1 study revealed clinically meaningful prevention of bleeds as well as superiority in comparison to other factor prophylaxis. Investigators observed that efanesoctocog alfa was well-tolerated with undetectable inhibitor development.
“The results from the pivotal XTEND-1 Phase 3 study demonstrate efanesoctocog alfa’s ability to reduce annualized bleeding rates, which supports its potential as a therapy with best-in-disease efficacy. We look forward to working closely with the FDA during the review process as we aim to bring this novel therapy to the hemophilia A community," Dietmar Berger, MD, PhD, Global Head of Development and Chief Medical Officer, Sanofi, stated.