Once-weekly Altuviiio (efanesoctocog alfa) is approved in Japan as a new class of factor VIII therapy for hemophilia A.

Altuviiio is indicated for control of bleeding tendency in patients with hemophilia A (factor VIII deficiency). Altuviiio was also recently approved by the Taiwan Food and Drug Administration for treatment of adults and children with hemophilia A on August 31, 2023.

Also referred to as efanesoctocog alfa, Altuviiio is the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing in adults and adolescents, and significantly reduces bleeds compared to prior factor VIII prophylaxis in adults and adolescents with severe hemophilia A. Altuviiio can be used for routine prophylaxis, on-demand treatment and control of bleeding episodes, and perioperative management of bleeding. The simple recommended dose of 50 IU/kg is intended for all patients and for different clinical scenarios.

The MHLW approval is based on positive data from patients with severe hemophilia A, including the pivotal XTEND-1 trial in adults and adolescents and data from the XTEND-Kids trial in children under 12 years of age. In the XTEND-1 study, once-weekly Altuviiio prophylaxis (50 IU/kg) met the primary endpoint, providing significant bleed protection for people with severe hemophilia A with a mean annualized bleeding rate (ABR) of 0.71 (95% CI: 0.52 - 0.97) and a median ABR of 0.00 (Q1, Q3: 0.00, 1.04). Altuviiio met the key secondary endpoint with a significant reduction of 77% in ABR versus prior factor VIII prophylaxis based on an intra-patient comparison (95% CI: 58%, 87%).