Basic Information
ID DDInter2014
Drug Type small molecule
Molecular Formula C24H26N4O3S
Molecular Weight 450.560
CAS Number 1260075-17-9
Description Mitapivat is a novel, first-in-class pyruvate kinase activator. It works to increase the activity of erythrocyte pyruvate kinase, a key enzyme involved in the survival of red blood cells. Defects in the pyruvate kinase enzyme in various red blood cells disorders lead to the lack of energy production for red blood cells, leading to lifelong premature destruction of red blood cells or chronic hemolytic anemia.[A245478] On February 17, 2022, the FDA approved mitapivat as the first disease-modifying treatment for hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, inherited disorder leading to lifelong hemolytic anemia.[L40533] Mitapivat has also been investigated in other hereditary red blood cell disorders associated with hemolytic anemia, such as sickle cell disease and alpha- and beta-thalassemia.[A245478]
ATC Classification G01AE10
IUPAC Name N-{4-[4-(cyclopropylmethyl)piperazine-1-carbonyl]phenyl}quinoline-8-sulfonamide
InChI XAYGBKHKBBXDAK-UHFFFAOYSA-N
Canonical SMILES O=C(N1CCN(CC2CC2)CC1)C1=CC=C(NS(=O)(=O)C2=CC=CC3=C2N=CC=C3)C=C1
Useful Links DrugBank ChemSpider Wikipedia ChEMBL ZINC
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