Sanofi's Novel Factor VIII Therapy Efanesoctocog Alfa Receives FDA Fast Track Designation for Hemophilia A

February 19, 2021 /BioValleyBIOON/ -- Sanofi recently announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to efanesoctocog alfa (formerly BIVV001, rFVIIIFc-VWF-XTEN) for the treatment of patients with hemophilia A. Efaneseoctocog alfa is a novel factor VIII therapy that operates independently of von Willebrand factor (vWF), providing near-normal factor activity levels for most of the week under a once-weekly prophylactic regimen.

August 2017, United StatesFDAThe European Commission (EC) granted orphan drug designation (ODD) to efanesoctocog alfa. Sanofi and Sobi have entered into a collaboration to develop and commercialize efanesoctocog alfa.

Fast Track Designation (FTD) aims to accelerate drug development and expedited review for serious diseases, addressing critical areas of unmet medical needs. When an investigational drug receives Fast Track designation, it means that pharmaceutical companies can engage withFDAEngage in more frequent interactions; if the relevant criteria are met after submission of the marketing authorization application, the product will be eligible for accelerated approval and priority review, as well as rolling review.

Efanesoctocog alfa has the potential to transform factor replacement therapy for patients with hemophilia A and represents a potential new class of factor VIII replacement therapies. The half-life of traditional factor VIII therapies is limited by the chaperone effect of von Willebrand factor (vWF), which is considered to restrict the duration that factor remains in the body. Efanesoctocog alfa is a novel fusion protein developed based on innovative Fc fusion technology, extending its circulation time in the bloodstream by adding a region of vWF and an XTEN® polypeptide.

Efanesoctocog alfa has the potential to provide near-normal bleeding protection for most of the week, while the increased half-life may allow prophylactic treatment dosing frequency to be reduced to once weekly.

In preclinical models of hemophilia A, efanesoctocog alfa demonstrated fourfold greater efficacy in hemostatic control compared with recombinant factor VIII (rFVIII), positioning it as a promising therapy to provide superior and broader protection against all types of bleeding episodes in patients with severe hemophilia A.

The ongoing Phase 3 XTEND-1 study is evaluating the safety and efficacy of efanesoctocog alfa in previously treated patients aged ≥12 years (n=150) with severe hemophilia A. XTEND-1 is an open-label, non-randomized interventional study with two parallel assignment groups. Subjects in the prophylaxis group receive weekly prophylactic treatment with efanesoctocog alfa at a dose of 50 IU/kg for 52 weeks. Subjects in the on-demand treatment group receive on-demand treatment with efanesoctocog alfa (50 IU/kg) for 26 weeks, followed by a switch to once-weekly prophylactic treatment with efanesoctocog alfa for 26 weeks.

Currently, efanesoctocog alfa is undergoing clinical studies, and its safety and efficacy have not yet been reviewed by any regulatory authority.(Bio Valley Bioon.com)

Original Source: Efanesoctocog alfa grantedFDA Fast Track Designation for treatment of hemophilia A