FDA grants priority review to efanesoctocog alfa for treatment of hemophilia A
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The FDA granted priority review to efanesoctocog alfa as prophylactic treatment for patients with hemophilia A.
Efanesoctocog alfa (Sobi, Sanofi) is a novel fusion protein designed to be given as once-weekly recombinant factor VIII replacement therapy to prevent bleeding. The FDA previously granted the investigational treatment breakthrough therapy designation for the treatment of 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,” Steve Pipe, MD, professor and director of the pediatric hemophilia and coagulation disorders program at University of Michigan, said in a Sanofi-issued 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,” Pipe added. “Such therapeutic benefits would represent important advances in unmet medical needs for people with hemophilia A and may transform the prophylactic treatment landscape.”
The FDA granted priority review based on pivotal data from the phase 3 XTEND-1 study.
Results of the trial, presented at this year’s International Society on Thrombosis and Haemostasis Congress, showed prophylactic efanesoctocog alfa offered significant improvements in physical health, pain and joint health 1 year after the start of treatment.
The trial met its primary endpoint by providing clinically meaningful improvement in bleeding protection, with a median annualized bleed rate of 0 (interquartile range, 0-1.04) for patients in Arm A of the study (prophylactic therapy) who received 52 weeks of prophylactic treatment with efanesoctocog alfa.
Patients who received prophylactic efanesoctocog alfa had an estimated mean annualized bleed rate reduction of 77% (P < .0001).
Nearly two-thirds of patients (64.7%) in Arm A did not experience any bleeds during the study, whereas 76.9% of patients in Arm B (on-demand therapy followed by prophylactic therapy) had no bleeds during the study’s prophylaxis period.
The FDA established a target action date of Feb. 28 for a decision on approval of the investigational agent.
References:
- FDA grants priority review to efanesoctocog alta for people with hemophilia A (press release). Available at: www.sanofi.com/en/media-room/press-releases/2022/2022-08-30-05-30-00-2506359. Published Aug. 30, 2022. Accessed Aug. 30, 2022.
- von Drygalski A, et al. Abstract LB 01.4. Presented at: International Society on Thrombosis and Haemostasis 2022 Congress; July 9-13, 2022; London.
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