[Comment] Mitapivat for non-transfusion-dependent thalassaemia

Mitapivat is a potential first-in-class drug for both α and β non-transfusion-dependent thalassaemia (NTDT). Thalassaemia is the most common hereditary red blood cell disorder and results in ineffective haematopoiesis and anaemia. Globally, the prevalence of α-thalassaemia is 22·6%, with rates exceeding 50% in southeast Asia, and 1·5% of the global population are carriers of β-thalassaemia.1,2 The classification has been simplified by dividing the disease into transfusion-dependent thalassaemia and NTDT.