Sanofi and Sobi’s efanesoctocog alfa has been recommended for approval by the European Medicines Agency’s human medicines committee as a once-weekly treatment for haemophilia A.
The treatment has been recommended to help treat and prevent bleeds and perioperative prophylaxis in haemophilia A and is a high-sustained factor VIII replacement therapy for patients of all ages and any disease severity.
Affecting around one in 5,000 male births annually, and more rarely in females, haemophilia A is a rare, lifelong genetic condition that occurs when the body does not produce enough factor VIII, a protein essential for blood clotting.
People living with the condition can experience bleeding, pain, irreversible joint damage and life-threatening haemorrhages.
The Committee for Medicinal Products for Human Use’s (CHMP) decision was supported by results from the pivotal phase 3 studies, XTEND-1 and XTEND-KIDS, which evaluated the efficacy and safety of efanesoctocog alfa in adults, adolescents and children for 52 weeks.
Efanesoctocoq alfa helps to provide children, adolescents and adults with normal to near-normal factor VIII activity levels of above 40% for a significant portion of the week, resulting in significantly improved protection from bleeds compared to existing factor VIII prophylaxis.
Results showed that once-weekly efanesoctocog alfa prophylaxis demonstrated significant bleed protection in those with severe haemophilia A of any age and showed substantial improvement in joint and physical health, pain and overall quality of life, with no factor VIII inhibitors observed.
Lydia Abad-Franch, chief medical officer and head of research, development and medical affairs at Sobi, commented: “[The] announcement marks a major milestone in haemophilia care and moves us one step closer to bringing efanesoctocog alfa to patients in the EU.”
She continued: “With the potential to significantly improve treatment outcomes and quality of life for people living with haemophilia A, we are excited about the positive impact this treatment could have around the world.”
The positive opinion from the CHMP will be submitted to the European Commission for a marketing authorisation decision.
In 2023, efanesoctocog alfa was first approved by the US Food and Drug Administration after the regulator previously grantedthe treatment breakthrough therapy designation in May 2022, fast track designation in 2021 and an orphan drug designation in 2017.
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, and Sanofi holds these rights in North America and all other regions worldwide excluding the Sobi territory.