Agios is a commercial-stage biopharmaceutical company focused on rare hereditary blood disorders, specifically hemolytic anemias. Its core asset is mitapivat, a first-in-class oral small molecule that activates pyruvate kinase (PK) enzymes in red blood cells, improving red blood cell metabolism and addressing the underlying cause of several hereditary hemolytic anemias. Mitapivat is approved and sold as PYRUKYND for PK deficiency in the U.S. and EU, and as AQVESME for thalassemia in the U.S. (launched January 2026), with EU approval pending. Agios sells directly in the U.S. through a specialty rare disease commercial infrastructure of roughly 40 sales professionals, using a single specialty pharmacy model and patient support services. Outside the U.S., Agios works through regional distribution partners in Europe and the GCC under revenue-sharing arrangements. The thalassemia launch represents the primary near-term growth driver, targeting roughly 4,000 initially addressable adult patients in the U.S., though the launch involves a REMS program and a 10–12 week lag from prescription to revenue. Beyond thalassemia, mitapivat is in Phase 3 development for sickle cell disease, with mixed trial results and an ongoing regulatory discussion with the FDA. Agios is also advancing tebapivat, a more potent PK activator in Phase 2 for lower-risk myelodysplastic syndromes and sickle cell disease, as well as two earlier-stage pipeline assets. Agios does not manufacture its own drugs and relies on contract manufacturers. The company also holds a residual 3% royalty on U.S. net sales of vorasidenib above $1B annually.