FDA Grants Priority Review for Sanofi’s BIVV001

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

The U.S. Food and Drug Administration has accepted for priority review the Biologics License Application (BLA) for Sanofi’s investigational therapy efanesoctocog alfa (BIVV001).

BIVV001 is a recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic intravenous 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 infusion. In contrast to other FVIII replacement therapies, it is independent of von Willebrand factor.

The BLA is supported by data generated from the XTEND-1 clinical trials, designed to evaluate the safety, efficacy, and pharmacokinetics of BIVV001 in previously treated adults and adolescents with hemophilia A, who are 12 years of age and older.

According to a new Sanofi press release, the study encompassed two parallel treatment arms — the prophylaxis Arm A in which patients who had received prior factor VIII prophylaxis were treated with once-weekly BIVV001 prophylaxis for 52 weeks, and the on-demand Arm B in which patients who had received prior on-demand factor VIII therapy began 26 weeks of on demand BIVV001, then switched to once-weekly prophylaxis for an additional 26 weeks. Trial data demonstrated “clinically meaningful” prevention of bleeds and superiority to prior factor prophylaxis based on an intra-patient comparison. The positive results were presented at the 2022 International Society on Thrombosis and Haemostasis Congress, held July 9-13th in London.

“The results from the pivotal XTEND-1 Phase 3 study demonstrate efanesoctocog alfa’s ability to reduce annualized bleeding rates, which supports its potential as a therapy with best-in-disease efficacy, said Dietmar Berger, Global Head of Development and Chief Medical Officer at Sanofi. “We look forward to working closely with the FDA during the review process as we aim to bring this novel therapy to the hemophilia A community.”

Sanofi also noted February 28, 2023, as the FDA decision date for BIVV001.

Source: Sanofi press release dated August 30, 2022