voxelotor

Voxelotor is a small molecule hemoglobin S (HbS) polymerization inhibitor developed for the treatment of sickle cell disease. It works by binding reversibly to the α-globin chains of hemoglobin, increasing its affinity for oxygen and stabilizing the oxygenated state. This prevents HbS polymerization, which is responsible for red blood cell sickling and subsequent vaso-occlusive crises in sickle cell disease. Voxelotor has been shown to increase hemoglobin levels, reduce markers of hemolysis (such as indirect bilirubin and reticulocytes), improve red blood cell deformability, and potentially decrease whole blood viscosity. The drug was approved for use in adults and children with sickle cell disease but was voluntarily withdrawn from the market in 2024 due to safety concerns[2][4][5][6][8].

Brand names

Oxbryta

Other names

voxelotorGBT440GBT-440GBT 440Oxbryta