Recombinant Factor VIII Fc for the Treatment of Haemophilia A

Authors: Hermans C; Mancuso, ME; Nolan, B; Pasi, KJ.

Affiliations: Haemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium; Center for Thrombosis and Hemorrhagic Diseases, Humanitas Clinical and Research Center – IRCCS, Rozzano, Italy; Children’s Health Ireland at Crumlin, Dublin, Ireland; Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, UK.

Publication: European Journal of Haematology; 2021 ; April 2021

Abstract: Prophylaxis with factor VIII (FVIII) is the current therapeutic approach for people with haemophilia A. However, standard half-life (SHL) FVIII products must be injected frequently, imposing a substantial burden on the individual and making it difficult to tailor therapy according to patient need and lifestyle, which could impact adherence. Recombinant FVIII Fc fusion protein (rFVIIIFc; Elocta®, Sobi; Eloctate®, Sanofi) is a recombinant fusion protein that undergoes slower clearance from the body than SHL FVIII products. This pharmacokinetic property of rFVIIIFc allows prophylactic administration every 3-5 days, or once weekly in selected patients, with doses adjusted to patient needs and clinical outcomes. Higher FVIII levels can be achieved maintaining dosing frequency similar to that usually applied with SHL FVIII. This review provides a summary of recent data from the A-LONG, Kids A-LONG, ASPIRE and PUPs A-LONG studies and recently published real-world experience relevant to rFVIIIFc use in individualised regimens. The review also introduces ongoing studies of rFVIIIFc, including its use for induction of immune tolerance, and discusses some aspects to consider when switching patients to rFVIIIFc and managing ongoing treatment. In summary, rFVIIIFc is suitable for individualised prophylaxis regimens that can be tailored according to patient clinical needs and lifestyle.