EU Approves Sobi's Weekly Haemophilia A Therapy Altuvoct

The European Commission has granted approval for Swedish Orphan Biovitrum’s (Sobi) Altuvoct (efanesoctocog alfa) for the treatment and prevention of bleeding episodes, as well as for perioperative prophylaxis in patients with haemophilia A. This approval comes after a favorable opinion from the European Medicines Agency's (EMA) drug advisory committee in April. Altuvoct, a once-weekly factor VIII replacement therapy, is designed for use in patients of all ages and various severities of haemophilia A. The therapy maintains non-haemophilia factor VIII levels above 40% for the majority of the week and achieves a trough level of 15% in adults and adolescents before the next dose.

Lydia Abad-Franch, Sobi's Chief Medical Officer, stated that this is the first instance where factor VIII activity levels can be sustained for a substantial part of the week with a simplified once-weekly dosing regimen. She highlighted the potential for this treatment to significantly improve both treatment outcomes and the quality of life for patients.

The extended approval of Altuvoct is based on data from two pivotal Phase III clinical trials – XTEND-1, which included adolescents and adults, and XTEND-Kids, which focused on children. The results of these studies demonstrated that a once-weekly administration of Altuvoct provides significant protection against bleeding across all age groups. Additionally, there were noted improvements in joint health, physical health, pain management, and overall quality of life over a 52-week period compared to baseline measurements. Importantly, no factor VIII inhibitors were observed in the Altuvoct clinical program.

In the previous year, the therapy received clearance in the United States and Japan, where it is marketed by Sanofi under the name Altuviiio. Following the positive results from the XTEND-1 trial, a Firstword survey of haematologists revealed that 73% of the respondents were highly impressed by the findings, with two-thirds indicating they would consider prescribing Altuviiio over Roche's Hemlibra (emicizumab) for newly diagnosed patients.

Meanwhile, Novo Nordisk is also a notable competitor in the haemophilia A market with its factor VIIIa mimetic bispecific antibody, Mim8. This treatment has recently shown potential for monthly dosing during the Phase IIIa FRONTIER 2 study, demonstrating another significant development in the treatment landscape for haemophilia A.

This new approval for Altuvoct represents a major advancement in haemophilia A treatment, offering a more manageable and effective option for patients, potentially transforming their therapeutic experience and overall quality of life.