The FDA approved Agios Pharmaceuticals’ mitapivat for the treatment of thalassemia under the brand name Aqvesme, a decision announced Dec. 24 after a short regulatory delay. The approval marks a new oral therapeutic option for patients with certain transfusion‑independent and dependent forms of thalassemia and expands the small‑molecule company’s commercial footprint. Mitapivat targets pyruvate kinase activators to improve red cell metabolism and reduce anemia; regulators authorized the drug based on clinical improvements in hemoglobin and transfusion requirements. The approval will affect treatment algorithms and payer negotiations for rare hematologic disorders and could reshape Agios’s near‑term commercial prospects.