mitapivat

Mitapivat is a first-in-class, oral small molecule that acts as an allosteric activator of erythrocyte pyruvate kinase (PKR), the enzyme responsible for the final step in glycolysis in red blood cells. By binding to a distinct allosteric site on PKR, mitapivat stabilizes its active tetrameric form and increases its affinity for phosphoenolpyruvate, leading to enhanced ATP production and reduced 2,3-diphosphoglycerate (2,3-DPG) levels. This mechanism addresses the underlying metabolic defect in patients with pyruvate kinase deficiency—a rare hereditary disorder causing chronic hemolytic anemia due to premature destruction of red blood cells. Mitapivat is approved for treating hemolytic anemia in adults with pyruvate kinase deficiency[1][4][7]. It also exhibits mild-to-moderate aromatase inhibition as an off-target effect[6].

Brand names

PyrukyndPyrukynd Taper Pack

Other names

mitapivat sulfate