French healthcare company Sanofi S.A. (Euronext Paris:SAN) (Nasdaq:SNY) said on Monday that the Japanese Ministry of Health, Labor and Welfare (MHLW) has granted marketing authorisation for ALTUVIIIO (Antihemophilic Factor [Recombinant], Fc-VWF-XTEN Fusion Protein), a high-sustained factor VIII replacement therapy.

ALTUVIIIO is designed for controlling bleeding tendency in individuals with haemophilia A (factor VIII deficiency).

The treatment was also recently approved by Taiwan's Food and Drug Administration for treatment of adults and children with haemophilia A.

ALTUVIIIO, also known as efanesoctocog alfa, is a groundbreaking treatment offering normal to near-normal factor activity levels (over 40%) for most of the week through once-weekly dosing in adults and adolescents. This approach significantly reduces bleeds compared to prior factor VIII prophylaxis in adults and adolescents with severe haemophilia A. ALTUVIIIO serves a range of purposes, including routine prophylaxis, on-demand treatment, control of bleeding episodes and perioperative management. The recommended dose of 50 IU/kg is applicable across all patients and clinical scenarios.

Haemophilia A is a rare lifelong condition characterized by impaired blood clotting, leading to excessive and spontaneous bleeds, especially into joints, resulting in joint damage, chronic pain, and potential impacts on quality of life. The severity of haemophilia correlates with clotting factor activity levels, with a higher risk of bleeding associated with lower factor activity.

The MHLW approval relies on positive data from severe haemophilia A patients, including the pivotal XTEND-1 trial in adults and adolescents and data from the XTEND-Kids trial in children under 12 years old.

Approved by the US Food and Drug Administration in February 2023, ALTUVIIIO previously received Breakthrough Therapy, Fast Track and Orphan Drug designations from the FDA. The European Commission granted Orphan Drug designation in June 2019, and the European Medicines Agency accepted the Marketing Authorisation Application (MAA) for efanesoctocog alfa in May 2023.

ALTUVIIIO is a result of collaboration between Sanofi and biopharmaceutical company Swedish Orphan Biovitrum AB (Sobi) (STO:SOBI), with final development and commercialisation rights divided between the two companies based on territories. Sobi covers Europe, North Africa, Russia and most Middle Eastern markets, while Sanofi handles North America and all other regions worldwide excluding the Sobi territory.