Mitapivat Improves Anaemia in Sickle Cell Disease: EHA 2026

MITAPIVAT significantly improved haemoglobin levels and markers of haemolysis in patients with sickle cell disease (SCD) in a Phase 3 trial, with additional clinical benefits observed among patients who achieved a haemoglobin response, a study presented at EHA 2026 congress found.

Mitapivat Targets Sickle Cell Anaemia

Persistent anaemia and ongoing haemolysis are major drivers of morbidity and mortality in SCD, contributing to acute complications, chronic organ damage and reduced quality of life. Mitapivat, an oral activator of pyruvate kinase in red blood cells, is designed to increase adenosine triphosphate (ATP) production while reducing levels of 2,3-diphosphoglycerate (2,3-DPG), potentially improving red blood cell health and reducing sickling. Investigators evaluated the efficacy and safety of the therapy in a global, randomised, double-blind, placebo-controlled Phase 3 trial involving 207 patients with SCD.