Efficacy and safety of mitapivat in adults with transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial

The authors aimed to evaluate the efficacy and safety of mitapivat, an oral allosteric activator of pyruvate kinase, in adults with transfusion-dependent α-thalassaemia or β-thalassaemia. The phase 3 trial found that mitapivat significantly reduced the transfusion burden compared to placebo, with a favorable safety profile, marking it as the first oral disease-modifying therapy for these conditions.