The FDA approved Agios Pharmaceuticals’ mitapivat under the brand name Aqvesme to treat certain forms of thalassemia, marking a key regulatory victory after a delayed review. The small‑molecule pyruvate kinase activator increases hemoglobin and red blood cell counts in affected patients. Approval gives Agios a commercial foothold in a rare‑disease hematology indication and validates mitapivat’s mechanism across hemoglobinopathies. Clinicians and payers will now evaluate labeling, patient selection criteria and access pathways for this oral therapy.