Phase 3 Trial Shows Mitapivat Efficacy and Safety for Transfusion-Dependent Thalassemia

Results from the double-blind, randomized, multicenter, placebo-controlled phase 3 ENERGIZE-T trial, published in The Lancet, demonstrate the efficacy and safety of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia. This study addresses a significant unmet need for disease-modifying therapies in these patient populations.