The US FDA has approved the first oral pill, mitapivat (Aqvesme), for adults with alpha- or beta-thalassemia, a historic milestone experts call a game changer. This first-in-class pyruvate kinase activator aims to improve red blood cell energy balance, potentially reducing transfusion dependence and complications. Health experts anticipate it will shift disease management towards more sustainable, patient-centered care by offering an easily administered oral option.