Following its recent US FDA approval, on this episode, Pip and Rich discuss mitapivat, an allosteric activator of red-cell pyruvate kinase. They focus on ENERGIZE, a randomised controlled trial of mitapivat vs placebo (2:1) randomisation in people living with transfusion dependent thalassaemia. It met its primary endpoint of achieving significantly more patients reaching a haemoglobin rise of at least 1 g/L. However, Pip and Rich discuss the relevance of this surrogate endpoint as well as the small effects on fatigue and quality of life.

Taher et al. Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial. Lancet. 2025 Jul 5;406(10498):33-42. doi: 10.1016/S0140-6736(25)00635-X.

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