Sanofi has successfully completed the Phase I trial for its von Willebrand disease (vWD) therapy, efanesoctocog alfa (BIV001), a novel antihemophilic factor recombinant fusion protein that acts as a coagulation factor VIII therapy. This is a significant potential addition to the future of the late stage vWD pipeline, which currently lacks novel molecules and therapies that can address severe vWD, says GlobalData, a leading data and analytics company.

vWD is a common bleeding disorder where the blood is unable to clot due to defects in the function of the von Willebrand factor (vWF), a protein that enables platelet adhesion.

Efanesoctocog alfa is a particularly promising therapy as it is designed to address type 2N and type 3 vWD, both of which have severe symptoms. The therapy is currently approved by the FDA for routine prophylaxis and on-demand treatment to control bleeding episodes and preoperative management (surgery) for both adults and children with hemophilia A under the brand name Altuviiio

Akash Patel, Pharma Analyst at GlobalData, comments: “The vWD late-stage pipeline currently has two therapies, vonvendi, a recombinant von Willebrand factor (vWF) that is currently undergoing Phase III trials for pediatric use, and BT-200, which is currently in Phase II for vWD type 2B and potentially a future competitor therapy for BIV001. A variety of therapies, such as BIV001, BT-200, and RvWF for severe vWD, will significantly improve the treatment paradigm for patients and create a market for vWD therapies that address specific subtypes.”

The completion of Phase I therapy by Sanofi presents significant competition to Guardian Therapeutics’s Phase II BT200 aptamer-based therapy as a coagulation factor VIII therapy already that has clinical evidence with its use in hemophilia A

Patel concludes: “Sanofi’s commercial reach as a big pharma company will ensure a large-scale launch across multiple markets simultaneously. This makes BIV001 a promising, although early-stage, therapy. Sanofi’s recent Phase I trial success will lead to increased competition in the vWD early-stage market and offer significant promise for a wave of therapies that address the more severe subtypes of vWD”.