ALTUVIIIO, also known as efanesoctocog alfa, is an antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein used for perioperative management of bleeding, controlling bleeding episodes, and routine prophylaxis.
The recommended dosage of ALTUVIIIO for all patients, and when used under different clinical scenarios is 50 IU/kg.
The company claimed that ALTUVIIIO is the first and only hemophilia A treatment that can deliver normal to near-normal factor activity levels (over 40%) for most of the week under once-weekly dosing regime in adults and adolescents.
It has also shown to significantly reduce bleeding compared with the previous factor VIII prophylaxis in adults and adolescents with severe hemophilia A.
The approval is based on the positive data from pivotal XTEND-1 trial in adults and adolescents, and XTEND-Kids trial in children aged below 12 years.
XTEND-1 study met the primary endpoint of bleed protection in hemophilia A patients who received ALTUVIIIO prophylaxis once a week with a mean annualised bleeding rate (ABR) of 0.71 and a median ABR of 0.00.
ALTUVIIIO also met the key secondary endpoint with a reduction of 77% in ABR compared with previous factor VIII prophylaxis.
A mean ABR of 0.6 and a median ABR of 0 was observed in the XTEND-Kids study of 73 children, aged below 12 years, who received once-weekly ALTUVIIIO for 52 weeks.
These studies also demonstrated a safety profile of ALTUVIIIO with no reports of factor VIII inhibitor development.
Last month, ALTUVIIIO received approval from the Taiwan Food and Drug Administration for treating hemophilia A in adults and children.
Sanofi executive vice president and Specialty Care ad interim global head Brian Foard said: “The approval of ALTUVIIIO in Japan and Taiwan represents a major step forward for people living with hemophilia A in those countries.”
Sanofi partnered with Sobi for the final development and commercialisation of ALTUVIIIO in the US.
In this collaboration, Sobi gained final development and commercialisation rights in the Sobi territory particularly Europe, North Africa, Russia and most Middle Eastern markets while Sanofi acquired development and acquisition rights in North America and all other regions excluding the Sobi territory.