The FDA approved Agios Pharmaceuticals’ mitapivat under the brand name Aqvesme for thalassemia, following a three‑month review delay and missed PDUFA deadline. The approval provides patients with an additional oral therapy option and represents a regulatory win for Agios after a prolonged review timeline. Agios’ label and launch plan will be watched for dosing, real‑world tolerability and payer coverage decisions. The approval is a commercial inflection for Agios’ hematology franchise and may prompt competitive positioning from other companies developing oral agents for inherited hemoglobinopathies.