FDA Grants Breakthrough Status to BIVV001

BIVV001 is an investigational recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic infusions.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of individuals with hemophilia A. Investigational breakthrough drugs are placed on a fast-track approval program and given intensive guidance from the FDA. Development and commercialization of BIVV001 is collaborative endeavor between Sanofi and Sobi®.

BIVV001 is a novel recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic infusions. It is manufactured through proprietary Fc fusion technologies to extend the half-life of the rFVIII, resulting in more sustained protection from bleeds per intravenous infusion. Unlike like other FVIII replacement therapies, it is independent of von Willebrand factor.

Breakthrough designation was ensured, in large part, based on results of the XTEND-1 phase 3 clinical trial data program. These data demonstrated that once-weekly infusions of BIVV001 resulted in a “clinically meaningful” prevention of bleeds in individuals with severe hemophilia A over a 52-week period. According to a Sanofi press release, it was shown to be superior to prior prophylactic FVIII replacement therapy in preventing bleeding events based on an intra-patient comparison.

In terms of safety, the therapy was well-tolerated, with no reports of FVIII inhibitor development. The most commonly reported treatment-emergent adverse events were headache, joint stiffness, falls, and back pain – these events occurred in less than 5% of trial participants.

“The Breakthrough Therapy designation highlights efanesoctocog alfa’s potential to transform treatment for people with hemophilia A by providing higher protection for longer duration, said John Reed, MD, PhD, Global Head of Research and Development at Sanofi. “This potential new class of factor VIII therapy represents how we are boldly advancing science to address unmet needs for the hemophilia community. We are excited to work with regulatory authorities during the filing and review of this innovative therapy.”

Source: Sanofi press release dated June 1, 2022