The agency’s decision data for the investigational factor VIII therapy from Sanofi and Soba is set for February 28, 2023.
The FDA accepted the biologics license application (BLA) for efanesoctocog alfa (BIVV001; Sanofi, Sobi) for the treatment of hemophilia A for priority review.
Efanesoctocog alfa is an investigational recombinant factor VIII therapy that is intended to extend protection from bleeding with once weekly dosing.
The target action date for the FDA decision is February 28, 2023.
“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,” Steve Pip, MD, professor and director of Pediatric Hemophilia and Coagulation Disorders Program at the University of Michigan, said in a statement.
The BLA is supported by results from the XTEND-1 phase 3 study, which were presented at the 30th International Society of Thrombosis and Haemostasis Congress. The findings showed a clinically meaningful prevention of bleeds and superiority to prior factor prophylaxis based on an intra-patient comparison.
Efanesoctocog alfa was generally well-tolerated, and inhibitor development factor VIII was not detected. The most common treatment-emergent adverse events were arthralgia, back pain, falls, and headaches.
“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,” Dietmar Berger, MD, PhD, global head of development and chief medical officer at Sanofi, said in the statement.
The primary efficacy endpoint was the annualized bleeding rate (ABR) in arm A. The key secondary endpoint was an intra-patient comparison of ABR during the efanesoctocog alfa weekly prophylaxis treatment period compared with the prior factor VIII prophylaxis ABR for individuals in arm A. Those individuals participated in a previous observational study, Study 242HA201/OBS16221.
The study was interventional, non-randomized, and open-label that assessed the efficacy, safety, and pharmacokinetics of efanesoctocog alfa at a once-a-week dosage in individuals aged 12 years and older with severe hemophilia A who were previously treated with factor VIII replacement therapy.
Investigators separated individuals into 2 parallel treatment arms: the prophylaxis arm A, where those who had received prior factor VIII prophylaxis were treated with efanesoctocog alfa prophylaxis once a week intravenously for 52 weeks, and the on-demand arm B, where those had received prior on-demand factor VIII therapy began 26 weeks of on-demand efanesoctocog alfa, switching to once-weekly prophylaxis for an additional 26 weeks.
Efanesoctocog alfa previously received breakthrough therapy designation from the FDA in May 2022 and is the first factor VIII therapy to receive this designation. Additionally, the FDA also granted it orphan drug designation in August 2017 and fast-track designation in February 2021.
Regulatory submission in the European Union will follow when data from the ongoing XTEND-Kids pediatric study are available. This is expected in 2023.
The European Commission granted efanesoctocog alfa orphan drug designation in June 2019.
FDA grants priority review to efanesoctocog alfa for people with hemophilia A. News release. Sanofi. August 30, 2022. Accessed August 30, 2022. https://www.news.sanofi.us/press-releases?item=137377