etavopivat

{
  "id": "MLC-T-ONC-013447",
  "term": "etavopivat",
  "field": "Oncology and biomedicine",
  "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.",
  "synonyms": [
    "pyruvate kinase-R activator FT-4202",
    "red cell pyruvate kinase agonist FT-4202",
    "pyruvate kinase isozyme R agonist FT-4202",
    "PK-R activator FT-4202",
    "red blood cell isoenzyme pyruvate kinase agonist FT-4202",
    "RBC isoenzyme PK activator FT-4202",
    "erythrocyte pyruvate kinase activator FT-4202"
  ],
  "references": [
    "National Cancer Institute Thesaurus"
  ],
  "url": "https://mlchart.com/terminology/oncology/etavopivat/"
}