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2000
Volume 3, Issue 1
  • ISSN: 2210-299X
  • E-ISSN: 2210-3007
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Abstract

Mitapivat (AG-348) is a novel, first-in-class oral small-molecule allosteric activator of the pyruvate kinase enzyme. Mitapivat has been shown to significantly upregulate both wild-type and numerous mutant forms of erythrocyte pyruvate kinase (PKR), increasing adenosine triphosphate (ATP) production and reducing levels of 2,3-diphosphoglycerate. Given this mechanism, mitapivat has been evaluated in clinical trials in a wide range of hereditary hemolytic anaemias, including pyruvate kinase deficiency (PKD), sickle cell disease, and thalassemia.

The technique entails searching numerous search engines, such as PubMed, Science Direct, and Sci Finder, for relevant citations to the current subject matter. This is done in order to obtain the data that is required. In relation to medicine, mitapivat has been examined for its ability to cure a wide variety of inherited haemolytic anaemias in clinical investigations. Some examples of these conditions include pyruvate kinase deficiency (PKD), sickle cell disease, and thalassemias.

It has been demonstrated that mitapivat is both safe and effective in treating adults with PKD in two phases III clinical trials, and the development of the medicine is very close to being finished. Based on these findings, mitapivat may end up becoming the very first medication in the history of the world to receive regulatory approval.

Allosteric activator of pyruvate kinase mitapivat has shown promise in treating various hereditary hemolytic anemias, including sickle cell disease, PKD, and alpha- and beta-thalassemia.

© 2025 The Author(s).
© 2025 The Author(s). Published by Bentham Science Publishers. This is an open access article published under CC BY 4.0 https://creativecommons.org/licenses/by/4.0/legalcode
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2025-01-01
2025-09-25

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Mitapivat: A Novel Drug Discovery for the Treatment of Hereditary Hemolytic Anaemias
Curr. Indian Sci. 3, e2210299X261752 (2025); https://doi.org/10.2174/012210299X261752230922114714
/content/journals/cis/10.2174/012210299X261752230922114714
/content/journals/cis/10.2174/012210299X261752230922114714
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  • Article Type:     Review Article
Keyword(s): Clinical trials; FDA; Haemolytic anaemia; Mitapivat; Pyruvate kinase deficiency; Sickle cell disease; Thalassemia

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