The target action date for the agency’s decision for efanesoctocog alfa, a factor VIII therapy, is Feb. 28, 2023.
The FDA has accepted for priority review the biologics license application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of hemophilia A, a rare bleeding disorder. The target action date for the FDA decision is February 28, 2023.
Sanofi and Sobi collaborated on the development and commercialization of efanesoctocog alfa. Efanesoctocog alfa is a novel recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.
“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,” Steven Pipe, M.D., professor and director of Pediatric Hemophilia and Coagulation Disorders Program, University of Michigan, said in a press release. “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.”
The BLA was based on the XTEND-1 phase 3 study, which found that efanesoctocog alfa demonstrated a clinically meaningful prevention of bleeds in people with severe hemophilia A over a 52-week period. Efanesoctocog alfa was able to deliver high sustained factor activity levels in the normal to near-normal range for the majority of the week with once weekly prophylaxis dosing.
The key secondary endpoint was also met, demonstrating that efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy in preventing bleeding events 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 were headache, arthralgia, fall, and back pain.