European Commission Approves Sobi®'s ALTUVOCT™ for Haemophilia A Treatment

The European Commission has granted Marketing Authorisation for ALTUVOCT™ (efanesoctocog alfa), a new treatment for haemophilia A. Announced by Sobi® on June 19, 2024, this high-sustained factor VIII replacement therapy is designed for individuals of all ages and levels of disease severity. ALTUVOCT allows patients to achieve non-haemophilia factor VIII activity levels (above 40%) for a significant portion of the week with just a once-weekly prophylaxis. This results in better protection from bleeds, with adults and adolescents reaching trough levels of 15% before their next dose.

The European Medicines Agency (EMA) has also endorsed ALTUVOCT's orphan drug designation, granting it a 10-year market exclusivity period. The EMA highlighted that despite existing treatments, the once-weekly ALTUVOCT prophylaxis significantly reduces the annual bleeding rate compared to other factor VIII products, marking it as a clinically relevant advancement.

Professor Robert Klamroth from the Vivantes Klinikum Friedrichshain in Berlin emphasized the potential of ALTUVOCT to improve quality of life for people with haemophilia A. According to him, trials showed substantial improvements in the prevention and treatment of bleeds, as well as significant enhancements in physical health, pain management, and joint health.

The Marketing Authorisation is based on the results of pivotal phase 3 studies: XTEND-1, involving adults and adolescents, and XTEND-Kids, focusing on children. These studies assessed the efficacy and safety of ALTUVOCT in severe haemophilia A patients. The results demonstrated that ALTUVOCT (50 IU/kg) once-weekly prophylaxis provided significant bleed protection across all ages, with mean annual bleeding rates (ABR) of less than 1 and 80-88% of patients being free from spontaneous bleeds. Additionally, there were noted improvements in joint health, physical health, pain, and overall quality of life from baseline to the 52-week mark. Importantly, no factor VIII inhibitors were observed in the ALTUVOCT clinical program.

Lydia Abad-Franch, Chief Medical Officer at Sobi, remarked on the significance of this approval for haemophilia care. She highlighted that for the first time, sustained factor VIII activity levels could be achieved for a significant portion of the week with a simple once-weekly dose, offering new possibilities for normal haemostasis.

Haemophilia A is a rare genetic disorder where the body either produces insufficient amounts of or dysfunctional factor VIII, a protein crucial for blood clotting. It mainly affects males, occurring in about one in every 5,000 male births annually. People with haemophilia A are prone to bleeding episodes that can lead to pain, irreversible joint damage, and potentially life-threatening haemorrhages. While treatment options have advanced over time, unmet clinical and social needs remain.

ALTUVOCT was first approved in the United States by the FDA in February 2023. The FDA had previously granted it Breakthrough Therapy designation in May 2022, Fast Track designation in February 2021, and Orphan Drug designation in 2017.

ALTUVOCT, also known as ALTUVIIIO™ in the United States, Japan, and Taiwan, is a pioneering therapy combining Fc-VWF-XTEN fusion protein. This innovative design extends the protein's time in circulation, breaking through the von Willebrand factor ceiling that limits the half-life of current factor VIII therapies. ALTUVOCT is marketed by Sobi in Europe and by Sanofi in other regions, highlighting their collaborative efforts in developing and commercializing advanced haemophilia treatments.

Haemophilia A affects individuals differently, and innovative treatments like ALTUVOCT are crucial in addressing their varied needs. The approval of ALTUVOCT signifies a major advancement in haemophilia care, promising improved treatment outcomes and quality of life for patients.