Rare enzyme activator, Mitapivat

Mitapivat is an allosteric activator of red blood cell pyruvate kinase, the enzyme that performs the final step of glycolysis: it catalyzes the conversion of phosphoenolpyruvate to pyruvate. Enzyme activators as drugs are very rare. After a Phase III clinical trial, it was approved by the FDA earlier this year as the first disease-modifying therapy for hemolytic anemia in adults with pyruvate kinase deficiency.