The FDA approved Agios Pharmaceuticals’ mitapivat for thalassemia under the brand name Aqvesme, marking a regulatory win after a three‑month review delay. The approval provides a new oral option for patients with certain forms of thalassemia and represents an important commercial milestone for Agios. Mitapivat’s approval follows a series of clinical trials showing improvements in hemoglobin and red blood cell parameters in thalassemia patients. Agios emphasized the significance of an oral, disease‑modifying therapy entering a field with limited medical options. The new label may influence prescribing patterns for inherited hemoglobinopathies and will likely be monitored by payers given the small‑population, specialty‑drug context.