Mitapivat, sold under the brand name Pyrukynd, is a medication used to treat hemolytic anemia.
It is taken as the sulfate hydrate salt by mouth.
It is a pyruvate kinase activator.
An oral agent.
Mitapivat is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.
It increases red cell oxygen affinity inducing and hypoxic drive sufficient to cause increases in hemoglobin levels.
It binds to and activates pyruvate kinase, thereby enhancing glycolytic pathway activity, improving adenosine triphosphate (ATP) levels and reducing 2,3-diphosphoglycerate (2,3-DPG) levels.
Mutations in pyruvate kinase cause deficiency in pyruvate kinase which prevents adequate red blood cell (RBC) glycolysis, leading to a buildup of the upstream glycolytic intermediate 2,3-DPG and deficiency in the pyruvate kinase product ATP.
May be efficacious in sickle cell disease.