etavopivat

Etavopivat is an orally available, small-molecule allosteric activator of the red blood cell (RBC) isoform of pyruvate kinase (PK-R, also known as PKR). It is under investigation for the treatment of sickle cell disease and thalassemia. By allosterically binding to and activating PK-R in erythrocytes, etavopivat enhances glycolytic activity in RBCs, leading to increased adenosine triphosphate (ATP) production and reduced levels of 2,3-diphosphoglycerate (2,3-DPG). This results in improved oxygen affinity of hemoglobin, better RBC deformability, decreased hemolysis of sickled cells, increased hemoglobin levels, and improved membrane function. The drug aims to address anemia and reduce vaso-occlusive crises by prolonging RBC survival and improving their physiological properties[1][2][3][4][6].

Other names

etavopivat [INN]etavopivat [USAN]2245053-57-8