The US Food and Drug Administration (FDA) has approved mitapivat, the first-ever oral pill to treat anaemia in adults with alpha and beta thalassemia, marking a historic breakthrough in managing the genetic blood disorder. The drug, to be marketed as Aqvesme, is indicated for both transfusion-dependent and non-transfusion-dependent patients.
Health experts say the approval could significantly reduce patients’ reliance on lifelong blood transfusions and chelation therapy. “Mitapivat can be a path-breaking drug for thalassemia management, as it offers a simple oral option that may lower transfusion needs,” said Dr. Satyam Arora of PGICH, Noida.
Beyond improving haemoglobin levels, the oral therapy is expected to reduce fatigue, treatment burden, and long-term complications, paving the way for more patient-centric care.
With India being home to nearly one-eighth of the world’s thalassemia patients, experts have expressed hope that early access to the drug could ease the country’s blood supply burden and improve quality of life for thousands of patients.
