FDA grants breakthrough therapy designation to efanesoctocog alfa for hemophilia A
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The FDA granted breakthrough therapy designation to efanesoctocog alfa for treatment of hemophilia A.
Efanesoctocog alfa (BIVV001; Sanofi, Sobi) is an investigational recombinant factor VIII therapy designed to extend protection from bleeds with once-weekly prophylactic dosing.
The FDA based the designation on results of the phase 3 XTEND-1 study.
Topline results showed the study met its primary endpoint, with efanesoctocog alfa resulting in a clinically meaningful prevention of bleeds among people with severe hemophilia A over a 52-week period, according to a Sanofi-issued press release.
The study also met its key secondary endpoint, as efanesoctocog alfa appeared superior to prior prophylactic factor VIII replacement therapy for preventing bleeding events based on an intra-patient comparison.
Efanesoctocog alfa appeared well-tolerated, with no detection of inhibitor development to factor VIII. The most common treatment-emergent adverse events included headache, arthralgia, falls and back pain.
Data from XTEND-1 are expected to be shared at a medical meeting.
“The breakthrough therapy designation highlights efanesoctocog alfa’s potential to transform treatment for people with hemophilia A by providing higher protection for longer duration,” John Reed, MD, PhD, global head of research and development at Sanofi, said in the release.
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