Sobi's Altuvoct approved in EU for haemophilia A therapy

Haemophilia A is a genetic disorder characterized by an impaired blood clotting ability due to a deficiency or absence of clotting factor VIII. The European Commission has recently granted marketing authorization to Sobi’s Altuvoct (efanesoctocog alfa) for treating and preventing bleeding episodes and for perioperative prophylaxis in individuals with haemophilia A. This decision aligns with the European Medicines Agency (EMA) recommendation to maintain Altuvoct's orphan drug designation, providing it with a ten-year market exclusivity, a notable increase from the typical eight years granted to new drugs.

This European authorization follows the earlier approval by the US Food and Drug Administration (FDA) in February, which allowed Altuvoct, marketed as Altuviiio in the US, to be used for routine prophylaxis, on-demand treatment of bleeding episodes, and perioperative management. The therapy is a high-sustained recombinant factor VIII replacement administered weekly.

Haemophilia A, affecting fewer than 1 in 10,000 people, is an X-chromosome-linked disorder predominantly seen in males. According to the Canadian Hemophilia Society, this condition is exceedingly rare in females. Altuvoct's approval by the European Commission was based on data from the Phase III XTEND-1 clinical trial involving adults and adolescents and from the XTEND-Kids trial, which included children under 12.

Sobi has co-developed and commercialized Altuvoct in collaboration with Sanofi. Sobi controls the final development stages and commercialization in Europe, North Africa, Russia, and most Middle Eastern markets, while Sanofi holds these rights in North America and other regions outside Sobi’s territory. GlobalData projects that Altuvoct will generate approximately $2.74 billion in sales by 2030.

An additional high-profile treatment option for haemophilia A is gene therapy, which, despite its potential, faces significant adoption barriers due to its high cost. In June 2023, the US FDA approved BioMarin’s gene therapy, Roctavian (valoctocogene roxaparvovec-rvox), specifically for adults with haemophilia A. However, the therapy's steep price of $2.9 million far surpassed experts' predictions, who had estimated the cost to be around $1.95 million.