Sobi has received marketing authorisation from the European Commission (EC) for the use of Altuvoct (efanesoctocog alfa) in haemophilia A patients.
The factor VIII replacement therapy, which is being jointly developed and commercialised with Sanofi, has been authorised to treat and prevent bleeds and perioperative prophylaxis in patients with the rare bleeding disorder, regardless of age or disease severity.
The EC has also endorsed the European Medicines Agency’s (EMA) recommendation to retain Altuvoct’s orphan drug designation, granting a ten-year market exclusivity period for the therapy.
Affecting approximately one in every 5,000 male births annually, haemophilia A is a genetic condition resulting from insufficient levels of functioning factor VIII, a protein that is essential for blood clotting.
Administered as an intravenous injection, Altuvoct has the potential to deliver near-normal factor activity levels for a large part of the week, improving bleed protection with a once-weekly dose. The therapy is already approved and marketed as Altuviiio by Sanofi in the US, Japan and Taiwan.
The EC’s decision follows a recent recommendation from the European Medicines Agency’s human medicines committee and is supported by positive results from the late-stage XTEND-1 and XTEND-KIDS trials, which evaluated the efficacy and safety of Altuvoct in adults, adolescents and children for 52 weeks.
Results demonstrated that a weekly dose of Altuvoct provided significant bleed protection for severe haemophilia A patients of any age, with substantial improvements also observed in joint health, physical health, pain and overall quality of life compared to baseline.
Lydia Abad-Franch, chief medical officer and head of research, development and medical affairs at Sobi, described the approval as a “major step forward in haemophilia care”.
“For the first time, factor VIII activity levels can be sustained for a significant part of the week with simplified once-weekly dosing,” she said.
Sanofi and Sobi collaborate on the development and commercialisation of efanesoctocog alfa in the US, while Sobi has final development and commercialisation rights in Europe, North Africa, Russia and most Middle Eastern markets, with Sanofi holding these rights in North America and all other regions worldwide, excluding the Sobi territory.