Sanofi's Weekly Once Hemophilia A Therapy Efanesoctocog Alfa Receives FDA Approval

On February 23, Sanofi announced that the FDA had approved the Biologics License Application (BLA) for efanesoctocog alfa, a first-in-class, high-persistence Factor VIII replacement therapy for the treatment of Hemophilia A. The FDA had previously granted Efanesoctocog alfa Fast Track designation as well as Orphan Drug designation.

Efanesoctocog Alfa (Brand Name: ALTUVIIIO) is a novel Factor VIII therapy that extends the half-life of Factor VIII by fusing it with Fc, a fragment of von Willebrand factor (vWF), and an XTEN polypeptide segment, overcoming the limitation imposed by vWF on Factor VIII's half-life extension. This significantly prolongs the drug’s presence in the bloodstream. The therapy aims to maintain normal Factor VIII activity levels through once-weekly prophylactic dosing, providing extended bleeding protection for patients with Hemophilia A.

This marketing application is based on data from an open-label, multicenter Phase III study (XTEND-1). The study enrolled 159 patients aged 12 years and older to evaluate the safety, efficacy, and pharmacokinetic profile of once-weekly efanesoctocog alfa in severe hemophilia A patients previously treated with Factor VIII (FVIII) replacement therapy.

XTEND-1 Design Scheme

XTEND-1 has two parallel groups: Group A, the prophylaxis group (n=133), received 50 IU/kg Efanesoctocog alfa weekly for 52 weeks; Group B, the on-demand group (n=26), initially received 50 IU/kg as needed for 26 weeks, then switched to once-weekly dosing for an additional 26 weeks.

The primary endpoint of the study was the annual bleeding rate (ABR) in Group A. The results showed that in Group A patients receiving once-weekly preventive treatment with efanesoctocog alfa, the median ABR over 52 weeks was 0, and the mean ABR was 0.70, achieving the primary endpoint of clinically meaningful bleeding prevention.

Compared with previous prophylactic FVIII replacement therapy, efanesoctocog alfa demonstrated superiority in preventing bleeding episodes. Efanesoctocog alfa can maintain FVIII levels within the normal to near-normal range for most of the week.

In addition, interim data from XTEND-Kids showed that children under 12 years old (n=23) receiving efanesoctocog alfa once weekly for 26 weeks had a mean ABR of 0.5 and a median ABR of 0.

Sanofi CEO Paul Hudson stated: "The approval of Efanesoctocog alfa today allows patients and doctors to redefine the lives of those living with hemophilia. With a once-weekly dose, Efanesoctocog alfa can sustainably achieve factor activity levels that have the potential to transform bleeding outcomes for people with hemophilia."

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