Preferred Label : Mitapivat;
NCIt synonyms : Red Cell Pyruvate Kinase Activator AG-348; PK-R Activator AG-348; PKR-IN-1;
NCIt 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.;
UNII : 2WTV10SIKH;
InChIKey : XAYGBKHKBBXDAK-UHFFFAOYSA-N;
CAS number : 1260075-17-9;
Molecule name : AG-348; AG 348;
NCI Metathesaurus CUI : CL936034;
Codes from synonyms : 58331;
Origin ID : C157039;
UMLS CUI : C5226639;
Currated CISMeF NLP mapping
- Semantic type(s)
- UMLS correspondences (same concept)
- Validated automatic mappings to BTNT
- concept_is_in_subset
- has_salt_form
- is_free_acid_or_base_form_of
