Sanofi and Sobi's Efanesoctocog Alfa Receives FDA Breakthrough Therapy Designation for Hemophilia A

Today, Sanofi announced that the U.S. FDA has granted breakthrough therapy designation to efanesoctocog alfa (BIVV001), which is being developed in collaboration with Sobi, for the treatment of patients with hemophilia A. The press release noted that this is the first factor VIII (FVIII) therapy to receive breakthrough therapy designation. Efanesoctocog alfa is a long-acting hemophilia A therapy aimed at maintaining near-normal FVIII activity for most of the week through once-weekly prophylactic treatment.

Hemophilia A is a hereditary disease caused by mutations in the gene encoding FVIII, resulting in the absence of FVIII. The current common treatment for hemophilia A is regular infusion of FVIII; however, patients typically require FVIII infusions 3-4 times per week, which brings significant inconvenience to their lives.

The half-life of natural FVIII in the blood is only about 12 hours because it forms a complex by binding with von Willebrand factor (VWF) in the blood. Although the binding of VWF increases the stability of FVIII in the blood, the degradation rate of VWF itself sets an upper limit on the retention time of FVIII in the blood, as when VWF is degraded, the FVIII bound to it is also degraded simultaneously.

BIVV001 is designed to link FVIII with a VWF fragment, forming a complex that does not bind with natural VWF in the blood, thereby overcoming the half-life ceiling set by VWF. It aims to enable hemophilia A patients to achieve near-normal FVIII activity levels for most of the week following a single injection.

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

The granting of this Breakthrough Therapy Designation is based on the positive results from the pivotal Phase 3 clinical trial XTEND-1. The trial results showed that weekly preventive treatment with efanesoctocog alfa significantly reduced the annual bleeding rate in patients with severe Hemophilia A. Additionally, efanesoctocog alfa demonstrated superiority in preventing bleeds compared to prior FVIII prophylactic therapy.

John Reed, MD, Global Head of Research and Development at Sanofi, said: "The Breakthrough Therapy Designation underscores the potential of efanesoctocog alfa to transform the treatment of hemophilia A, providing higher protection over a longer duration. We look forward to continuing our collaboration with regulatory authorities throughout the submission and review process for this innovative therapy."

References:

[1] Press Release: FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A. Retrieved June 1, 2022, from https://www.globenewswire.com/news-release/2022/06/01/2453803/0/en/Press-Release-FDA-grants-efanesoctocog-alfa-Breakthrough-Therapy-designation-for-hemophilia-A.html

[2] Konkle et al., (2020). BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A. NEJM, DOI: 10.1056/NEJMoa2002699

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