Preferred Label : Etavopivat;
NCIt synonyms : Pyruvate Kinase Isozyme R Agonist FT-4202; Red Cell Pyruvate Kinase Agonist FT-4202; Erythrocyte Pyruvate Kinase Activator FT-4202; PK-R Activator FT-4202; RBC Isoenzyme PK Activator FT-4202; Red Blood Cell Isoenzyme Pyruvate Kinase Agonist FT-4202; PKR Activator FT-4202; Pyruvate Kinase-R Activator FT-4202;
NCIt definition : An orally available, small-molecule allosteric activator of the selective red blood
cell (RBC) isoform of pyruvate kinase (PK-R), with potential to improve symptoms in
sickly cell disease (SCD) patients. Upon oral administration, etavopivat allosterically
binds to and activates PK-R, thereby enhancing the glycolytic pathway activity in
RBCs. This improves adenosine triphosphate (ATP) levels and reduces 2,3-diphosphoglycerate
(2,3-DPG) levels in RBCs. This results in increased oxygen affinity, improved RBC
deformability, decreased sickle RBC hemolysis, increased hemoglobin (Hb) levels and
improved RBC membrane function. Mutations in PK-R cause deficiency in PK-R which prevents
adequate RBC glycolysis, leading to a buildup of the upstream glycolytic intermediate
2,3-DPG and deficiency in the PK-R product ATP.;
UNII : V4E0A9M44Q;
CAS number : 2245053-57-8;
Molecule name : FT 4202; FT-4202;
NCI Metathesaurus CUI : CL1642933;
Origin ID : C176893;
UMLS CUI : C5554583;
Semantic type(s)
- concept_is_in_subset
