Sanofi and Swedish Orphan Biovitrum (Sobi) have announced positive results from a phase 3 trial, showing once-weekly efanesoctocog alfa provided ‘clinically meaningful’ bleed protection for patients with severe haemophilia A.
The data was presented for the first time on 10 July in a late-breaking session at the 30th International Society on Thrombosis and Haemostasis (ISTH) Congress.
Data from the XTEND-1 phase 3 study showed adults and adolescents 12 years and older treated once-weekly with efanesoctocog alfa experienced ‘statistically significant and clinically meaningful’ improvements in physical health, pain intensity and joint health.
Crucially, the study showed efanesoctocog alfa was effective at treating bleeds, including in target joints, with 96.7% of bleeds resolved with a single 50 IU/kg dose.
Commenting on the phase 3 trial results, Dietmar Berger, global head of development and chief medical officer at Sanofi, said: “We are committed to advancing innovative medicines that disrupt the status quo and address the unmet needs that persist for people with rare conditions like haemophilia. This robust data illustrates the promise of efanesoctocog alfa’s efficacy with once-weekly dosing and underscores its potential as a therapy with best-in-disease efficacy.”
Haemophilia A is a rare, genetic condition that affects the blood’s ability to clot. It occurs in around one in 5,000 male births annually, and more rarely in females. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.
Efanesoctocog alfa (formerly BIVV001) is a novel and investigational recombinant factor VIII therapy designed to extend protection from bleeds with once-weekly dosing. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
Anders Ullman, head of R&D and chief medical officer at Sobi, said: “We believe transforming the treatment paradigm for haemophilia A can only be achieved through elevating standards of care towards normal haemostasis. This data demonstrates the profile of efanesoctocog alfa in significant clinical terms, and further strengthens its potential to ultimately improve the lives of many living with this condition.”
The treatment was granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA) in May 2022, having received Fast Track designation in February 2021 and Orphan Drug designation in August 2017. The European Commission also granted efanesoctocog alfa Orphan drug designation in June 2019.