Mitapivat
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. Mitapivat is a pyruvate kinase activator.Mitapivat was approved for medical use in the United States in February 2022.

Medical uses
Mitapivat is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

Pharmacology
Mechanism of action
Mitapivat 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.

Society and culture
Legal status
On 15 September 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Pyrukynd, intended for the treatment of an inherited condition called pyruvate kinase deficiency. The applicant for this medicinal product is Agios Netherlands B.V.

Names
Mitapivat is the international nonproprietary name (INN).

References
Further reading
Kung C, Hixon J, Kosinski PA, Cianchetta G, Histen G, Chen Y,  et al. (September 2017). "AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency". Blood. 130 (11): 1347–1356. doi:10.1182/blood-2016-11-753525. PMC 5609468. PMID 28760888.
Rab MA, Van Oirschot BA, Kosinski PA, Hixon J, Johnson K, Chubukov V,  et al. (January 2021). "AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes". Haematologica. 106 (1): 238–249. doi:10.3324/haematol.2019.238865. PMC 7776327. PMID 31974203.

External links
"Mitapivat sulfate". Drug Information Portal. U.S. National Library of Medicine.
Clinical trial number NCT03548220 for "A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)" at ClinicalTrials.gov
Clinical trial number NCT03559699 for "A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)" at ClinicalTrials.gov