Preferred Label : Tebapivat;
NCIt synonyms : Pyruvate Kinase-R Activator AG-946; PKR Activator AG-946; PK-R Activator AG-946;
NCIt definition : An orally available activator of the red cell isoform of pyruvate kinase (PK-R; PKR),
with potential to improve hemolytic anemia and related-symptoms in patients with pyruvate
kinase deficiency (PKD). Upon oral administration, tebapivat binds to and activates
PKR, thereby enhancing glycolytic pathway activity in red blood cells (RBCs), improving
adenosine triphosphate (ATP) levels and reducing 2,3-diphosphoglycerate (2,3-DPG)
levels. This may result in increased oxygen affinity, improved RBC deformability,
decreased sickle RBC hemolysis, increased hemoglobin (Hb) levels and improved RBC
membrane function. Mutations in PKR cause deficiency in PKR which prevents adequate
RBC glycolysis, leading to a build-up of the upstream glycolytic intermediate 2,3-DPG
and deficiency in the PKR product ATP.;
UNII : ORG4KGP5ZS;
Molecule name : AG 946; AG-946;
NCI Metathesaurus CUI : CL1921995;
Origin ID : C203234;
UMLS CUI : C5909108;
Semantic type(s)
- concept_is_in_subset
