Agios Pharmaceuticals, Inc. is a biopharmaceutical firm dedicated to the discovery and advancement of innovative therapies focused on cellular metabolism for rare genetic conditions. The company offers PYRUKYND (mitapivat), an activator of pyruvate kinase enzymes, approved for treating hemolytic anemia in adults with pyruvate kinase deficiency. PYRUKYND is currently under U.S. FDA review for thalassemia, with a Prescription Drug User Fee Act goal date in early December 2025, and has already received a positive CHMP opinion in Europe and approval in Saudi Arabia for this indication. Additionally, results from its Phase 3 RISE UP trial of mitapivat in sickle cell disease demonstrated a successful hemoglobin response, though it did not achieve statistical significance in reducing pain crises. Agios' pipeline also includes Tebapivat, a PK activator in Phase 2 for MDS-associated anemia and sickle cell disease, and AG-946, a novel PK activator in Phase 1 for hemolytic anemias and other indications. Established in 2007, Agios Pharmaceuticals, Inc. is headquartered in Cambridge, Massachusetts.