Sanofi and Sobi Announce Long-Acting Hemophilia A Therapy Hits Key Phase 3 Endpoint

On March 10, Sanofi and Sobi jointly announced a pivotal Phase 3 clinical trial of their co-developed long-acting hemophilia A therapy efanesoctocog alfa (BIVV001) in patients over 12 years of age with severe hemophilia A positive top-line results

Hemophilia A is a genetic disorder in which FVIII is missing due to mutations in the gene encoding FVIII

The half-life of native FVIII in the blood is only about 12 hours, because in the blood it binds to von Willebrand factor (VWF) to form a complex

The design of BIVV001 links FVIII to a VWF fragment, and the resulting complex does not bind to native VWF in the blood, thus breaking the upper half-life limit set by VWF

▲Schematic diagram of the structure of BIVV001 (Image source: Reference [2])

This open-label, non-randomized Phase 3 clinical trial was conducted in 159 patients over 12 years of age with severe hemophilia

References:

[1] Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in hemophilia A, demonstrating superiority to prior factor prophylaxis treatment.

[2] Konkle et al.

(Original abridged)