Sobi Gains EC Approval for Altuvoct in Haemophilia A

Sobi has achieved a significant milestone with the European Commission (EC) granting marketing authorisation for Altuvoct (efanesoctocog alfa) to treat haemophilia A. This factor VIII replacement therapy, co-developed and commercialised with Sanofi, is designed to manage and prevent bleeding episodes as well as provide perioperative prophylaxis in patients suffering from this rare bleeding disorder, regardless of their age or the severity of their condition.

The EC's approval also includes the retention of Altuvoct's orphan drug designation, as recommended by the European Medicines Agency (EMA), which secures a decade of market exclusivity for the treatment. Haemophilia A, a genetic disorder that affects about one in every 5,000 male births each year, is characterized by inadequate levels of functioning factor VIII, a critical protein for blood clotting.

Altuvoct is administered via intravenous injection and has the potential to maintain nearly normal factor activity levels for most of the week with a single weekly dose. This enhances protection against bleeding episodes. The therapy is already marketed as Altuviiio by Sanofi in the US, Japan, and Taiwan.

The EC's decision was influenced by a favorable recommendation from the European Medicines Agency’s human medicines committee and is substantiated by positive results from the XTEND-1 and XTEND-KIDS trials. These late-stage studies, which spanned 52 weeks, assessed Altuvoct's efficacy and safety in adults, adolescents, and children with severe haemophilia A. The trials demonstrated that a weekly dose of Altuvoct provided significant protection against bleeding and led to notable improvements in joint health, physical health, pain management, and overall quality of life when compared to baseline conditions.

Lydia Abad-Franch, Sobi's chief medical officer and head of research, development, and medical affairs, hailed the approval as a transformative advancement in haemophilia care. She emphasized that, for the first time, factor VIII activity levels could be sustained for a significant portion of the week with a simplified once-weekly dosing regimen.

The development and commercialisation of efanesoctocog alfa are a collaborative effort between Sanofi and Sobi. In the United States, the two companies jointly manage these activities, while Sobi has the final development and commercialisation rights in Europe, North Africa, Russia, and most markets in the Middle East. Sanofi retains these rights in North America and other global regions, excluding the territories covered by Sobi.

This approval marks a significant step forward, offering a new, effective treatment option that promises enhanced quality of life for haemophilia A patients. The once-weekly administration simplifies the management of this chronic condition, potentially transforming the standard of care and providing much-needed relief to those affected.