USFDA grants FTD to efanesoctocog alfa for hemophilia A treatment

The U.S. Food and Drug Administration (FDA) has granted efanesoctocog alfa – earlier termed BIVV001 (rFVIIIFc-VWF-XTEN) – Fast Track Designation (FTD) for the treatment of patients suffering from hemophilia A.

Fast Track Designation is a process for facilitating the development and expediting the review of drugs to treat serious medical conditions.

The novel and investigational factor VIII therapy efanesoctocog alfa has been designed in such a way as to provide near-normal factor activity levels for most of the week in the once-weekly prophylactic treatment regimen.

Independent of the von Willebrand Factor, the drug’s unique make can potentially extend bleed protection in the once-weekly dose.

The USFDA granted efanesoctocog alfa orphan drug designation in August 2017, while the European Commission did the same in June 2019.

Global biopharmaceutical companies Sanofi and Sobi are collaborating on the development and commercialisation of efanesoctocog alfa.

Sobi’s territory for final development and commercialisation rights comprises Europe, Russia, North Africa and most Middle Eastern markets, while Sanofi’s territory comprises North America and all other regions in the world excluding Sobi’s territory.

Efanesoctocog alfa represents a new class of factor VIII replacement therapies and can potentially transform factor replacement therapy for patients suffering from hemophilia A.

The half-life of conventional factor VIII therapy is repressed by the von Willebrand Factor’s (VWF) chaperone effect, which is deemed to restrict the factor’s duration in the body.

Efanesoctocog alfa develops upon the Fc fusion technology by including a region of von Willebrand factor and XTEN polypeptides to prolong its circulation time.

Although Fc fusion technology has been in use for over 15 years, Sanofi and Sobi have augmented the technology to become the first companies to employ it for treating patients with hemophilia.

Efanesoctocog alfa’s safety and efficacy is currently under evaluation in the ongoing Phase 3 XTEND-1 study.

XTEND-1 is a non-randomised, open-label interventional study having two parallel assignment arms — the prophylaxis arm and the on-demand arm.