Etavopivat

**Semantic type:** Pharmacologic Substance

**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:** - ETAVOPIVAT - Erythrocyte Pyruvate Kinase Activator FT-4202 - FT 4202 - FT-4202 - FT4202 - PK-R Activator FT-4202 - PKR Activator FT-4202 - Pyruvate Kinase Isozyme R Agonist FT-4202 - Pyruvate Kinase-R Activator FT-4202 - RBC Isoenzyme PK Activator FT-4202 - Red Blood Cell Isoenzyme Pyruvate Kinase Agonist FT-4202 - Red Cell Pyruvate Kinase Agonist FT-4202