AGIOS PHARMACEUTICALS, INC.

AGIOS PHARMACEUTICALS, INC. is a commercial-stage biopharmaceutical company focused on rare disease treatment, with a foundation in hematology. The company develops and delivers innovative medicines. Its lead product candidate, mitapivat, activates both wild-type and mutant pyruvate kinase (PK) enzymes to treat hemolytic anemias. Mitapivat is approved in the United States under the brand name AQVESME™ for adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia. In Saudi Arabia, it is approved under the brand name PYRUKYND® for adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. Mitapivat is also approved as PYRUKYND® in the United States by the FDA for treating hemolytic anemia in adults with PK deficiency, and in the European Union and Great Britain for treating PK deficiency in adults. In December 2024, the company submitted a marketing authorization application to the European Medicines Agency (EMA) and a regulatory application to the United Arab Emirates health authorities for PYRUKYND® for adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia. In October 2025, the EMA's Committee for Medicinal Products for Human Use adopted a positive opinion for PYRUKYND® for this new indication in adults. The European Commission is reviewing this opinion, with a final decision anticipated in early 2026. The company plans a pre-supplemental New Drug Application meeting with the FDA in the first quarter of 2026.