Sanofi, Sobi announce results of XTEND-1 phase 3 study of efanesoctocog alfa
The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with efanesoctocog alfa over a period of 52 weeks.;
Paris: Sanofi and Swedish Orphan Biovitrum AB (publ) today announced positive topline results from the pivotal XTEND-1 Phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ≥12 years of age with severe hemophilia A.
The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with efanesoctocog alfa over a period of 52 weeks. The median annualized bleeding rate (ABR) was 0 with a mean ABR of 0.71. The key secondary endpoint was also met, demonstrating once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy, showing a statistically significant reduction in ABR based on 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.
Dietmar Berger, MD, PhD
Global Head of Development and Chief Medical Officer, Sanofi
"While advances have been made in the treatment of hemophilia, unmet medical needs still exist. These positive topline data, showing a very low annualized bleeding rate, enhance efanesoctocog alfa's potential to transform hemophilia A therapy. We believe efanesoctocog alfa provides higher protection for longer duration with reduced treatment burden of once-weekly dosing, and we look forward to working with regulators to bring this therapy to patients as soon as possible."
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