The U.S. Food and Drug Administration has approved mitapivat, the first oral medication designed to treat anemia in adults with alpha- or beta-thalassemia. This drug functions by enhancing red blood cell energy production, thereby increasing their lifespan and improving hemoglobin levels. For decades, patients have relied on blood transfusions, which carry risks like iron overload. Mitapivat offers a new treatment option for both transfusion-dependent and non-transfusion-dependent forms of the disease, marking a significant advancement for managing this inherited blood disorder.