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Mitapivat is a novel, first-in-class pyruvate kinase activator. It works as a small-molecule allosteric activator of the red blood cell-specific form of pyruvate kinase (PK-R), enhancing the activity of both wild-type and mutant enzymes associated with hemolytic anemia. By increasing the activity of erythrocyte pyruvate kinase, a key enzyme involved in red blood cell energy production, mitapivat addresses the underlying molecular pathology in pyruvate kinase deficiency, which leads to premature destruction of red blood cells and chronic hemolytic anemia.
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