FDA Approves First Oral Pill for Thalassemia-Related Anaemia

In a historic breakthrough, the US Food and Drug Administration has approved the world’s first oral pill for treating anaemia in adult thalassemia patients. The drug, mitapivat (Aqvesme), is approved for both transfusion-dependent and non-dependent alpha and beta thalassemia. By improving red blood cell survival at the cellular level, it can reduce reliance on lifelong blood transfusions. Experts have termed it a game-changer with major implications for global and Indian thalassemia care.