Abstract

REVIEW: A NOVEL DRUG MITAPIVAT (AG 348) IS PYRUVATE KINASE ACTIVATOR FOR THE MANAGEMENT OF HEMOLYTIC ANEMIA

Shubham R. Supekar*, Manoj H. Shinde, Mahadeo L. Ghule, Anuradha A. Patil and Vishal Babar

ABSTRACT

Mitapivat (AG-348) is a novel, first-in-class oral small molecule allosteric activator of red blood cell pyruvate kinase enzyme. Mitapivat has been shown to significantly, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes.and reducing levels of 2,3 diphosphoglycerate. The convenient oral dosing and a safety profile comparable with placebo in adults with Pyruvate kinase activator, mitapivat is a promising novel therapeutic for several hereditary hemolytic anemias, including those without any currently US Food and Drug Administration (FDA) or European Medicines Agency (EMA) approved drug therapies. Preclinical studies of mitapivat in thalassemia and sickle cell disease have also been performed.

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