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Plain language summary of efanesoctocog alfa for patients with severe hemophilia A

Publicated to:Future Rare Diseases. 4 (1): - 2024-01-01 4(1), DOI: 10.2217/frd-2023-0024

Authors: Drygalski Av; Chowdary P; Kulkarni R; Susen S; Konkle BA; Oldenburg J; Matino D; Klamroth R; Weyand AC; Jimenez-Yuste V; Nogami K; Winding B; Willemze A; Knobe K

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Abstract

What is this summary about?: This is a plain language summary of a clinical research study called XTEND-1. The study looked into how safe and effective a medicine called efanesoctocog alfa is for people with severe hemophilia A. Hemophilia A is a genetic condition in which the body does not produce enough or makes dysfunctional clotting factor VIII (eight)–a protein that is essential for blood clotting. People with hemophilia A are prone to dangerous bleeding, particularly internally and into their joints and muscles. How was the research done?: The XTEND-1 study compared two ways of using the medication to treat bleeding episodes: (1) injecting it once a week for 12 months as a prophylaxis (regular preventative) treatment, or (2) injecting it as needed for 6 months, followed by weekly prophylaxis treatment for a further 6 months. What did the research find out?: People who had prophylaxis treatment with efanesoctocog alfa injections for one year saw a significant reduction in the amount of bleeding they experienced compared to their pre-study prophylaxis treatment. The effects of the treatment also lasted longer, with higher factor VIII levels for longer, than previous prophylaxis treatment and less frequent injections were needed. There was a significant reduction in the amount of bleeding in people taking once-weekly prophylaxis treatment compared to ‘as needed’ treatment. Clinical Trial Registration: NCT04161495 (ClinicalTrials.gov).

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