Mitapivat

Mitapivat is a small molecule allosteric activator of pyruvate kinase (PK) enzymes, specifically the PK-R isozyme, which is predominantly expressed in red blood cells. It is used in the treatment of pyruvate kinase deficiency (PKD), a rare inherited hemolytic anemia.

Mitapivat works by binding to PK-R, increasing the enzyme's affinity for its substrates, phosphoenolpyruvate (PEP) and adenosine diphosphate (ADP). This activation of PK-R leads to an increase in the rate of glycolysis in red blood cells, improving ATP production and reducing the accumulation of 2,3-diphosphoglycerate (2,3-DPG). By improving red blood cell metabolism, mitapivat helps to reduce the severity of anemia in patients with PKD.

Mitapivat is administered orally. Common side effects can include fatigue, headache, and nausea.

It is marketed under the brand name Pyrukynd.