The FDA has accepted for priority review the Biologics License Application for efanesoctocog alfa for the treatment of hemophilia A.
Sanofi and Sobi collaborate on the development and commercialization of efanesoctocog alfa.
“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. Such therapeutic benefits would represent important advances in unmet medical needs for people with hemophilia A and may transform the prophylactic treatment landscape,” says Steve Pipe, MD, Professor and Director of Pediatric Hemophilia and Coagulation Disorders Program, University of Michigan.
The first factor VIII therapy to receive Breakthrough Therapy designation from the FDA
Efanesoctocog alfa received Breakthrough Therapy designation from the FDA in May 2022 and it is the first factor VIII therapy to receive this recognition. The FDA also granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.
Regulatory submission in the EU will follow availability of data from the ongoing XTEND-Kids pediatric study, with both events expected in 2023. The European Commission granted efanesoctocog alfa Orphan Drug designation in June 2019.
Data from the XTEND-1 Phase 3 study
The BLA is supported by data from the XTEND-1 Phase 3 study. The data demonstrate a clinically meaningful prevention of bleeds and superiority to prior factor prophylaxis based on an intra-patient comparison. Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected. The most common treatment-emergent adverse events (>5% of participants overall) were headache, arthralgia, fall, and back pain.