Mitapivat

**Semantic type:** Pharmacologic Substance

**Definition:** An orally available, small-molecule, allosteric activator of the red cell isoform of pyruvate kinase (PK-R), with potential to improve hemolytic anemia in patients with pyruvate kinase deficiency (PKD). Upon oral administration, mitapivat binds to and activates PK-R, thereby enhancing glycolytic pathway activity, improving adenosine triphosphate (ATP) levels and reducing 2,3-diphosphoglycerate (2,3-DPG) levels. Mutations in PK-R cause deficiency in PK-R which prevents adequate red blood cell (RBC) glycolysis, leading to a buildup of the upstream glycolytic intermediate 2,3-DPG and deficiency in the PK-R product ATP.

**Synonyms:** - AG 348 - AG-348 - AG348 - MITAPIVAT - PK-R Activator AG-348 - PKR-IN-1 - Red Cell Pyruvate Kinase Activator AG-348