Hemoglobin S (HbS) polymerization inhibitor Treatment of sickle cell disease

Sickle cell disease (SCD) is a hematological disorder of genetic origin, involving patients of every age group, and is characterized by altered hemoglobin structure resulting in polymerization of hemoglobin under hypoxic conditions. Current drugs approved for SCD are hydroxyurea and L-glutamine. Voxelotor is the latest drug approved for the management of SCD, along with crizanlizumab-tmca, by the U.S. Food and Drug Administration (FDA). Voxelotor has been shown to inhibit polymerization of sickle hemoglobin (HbS) and is claimed to have disease-modifying potential. In the clinical trials, it has been found to increase hemoglobin and decrease markers of hemolysis. This article mainly focuses on the pharmacology of voxelotor with special emphasis on relevant clinical trials and adverse drug reactions.