Mitapivat

Definition

An orally available, small-molecule, allosteric activator of the red cell isoform of pyruvate kinase (PK-R), with potential to improve hemolytic anemia in patients with pyruvate kinase deficiency (PKD). Upon oral administration, mitapivat binds to and activates PK-R, thereby enhancing glycolytic pathway activity, improving adenosine triphosphate (ATP) levels and reducing 2,3-diphosphoglycerate (2,3-DPG) levels. Mutations in PK-R cause deficiency in PK-R which prevents adequate red blood cell (RBC) glycolysis, leading to a buildup of the upstream glycolytic intermediate 2,3-DPG and deficiency in the PK-R product ATP.

Also known as AG 348, AG-348, AG348, MITAPIVAT, PK-R Activator AG-348, PKR-IN-1, Red Cell Pyruvate Kinase Activator AG-348 — per NCIT

Also identified as

• ATC B06AX04 per RXNORM
• NCIT NCIT:C157039 per NCIT
• SCTID 44102311000001100 per RXNORM
• UNII 2WTV10SIKH per RXNORM

Indications