The Biologics License Application (BLA) for Efanesoctocog alfa, a drug to treat the rare and life-threatening blood disease, hemophilia A, has been accepted for priority review by the U.S. Food and Drug Administration (FDA).
Efanesoctocog alfa (BIVV001) is being developed and commercialized as part of a collaboration between Sanofi and Sobi was given the target action date for the FDA’s decision for February 28, 2023.
Steve Pipe is a professor and director of pediatric hemophilia and coagulation disorders program, at the University of Michigan. He said: “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.”
The BLA is supported by data from the pivotal XTEND-1 phase 3 study. Results were recently presented at the 30th International Society of Thrombosis and Haemostasis Congress. 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 which was less than 5% of participants overall were headache, arthralgia, fall, and back pain.
Dietmar Berger chief medical officer at Sanofi, said: “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.”
The FDA grants priority review to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. 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.