The two medications in regular use for sickle cell disease are hydroxyurea and L-glutamine. Inhibiting HbS polymerization in red cells can have a disease-modifying effect.

The authors of this study aimed to evaluate the efficacy and safety of voxelotor, as compared with placebo, in adolescents and adults with sickle cell disease. A total of 274 patients were enrolled and randomly assigned in a 1:1:1 ratio to receive a once-daily oral dose of 1500mg of voxelotor, 900 mg of voxelotor, or placebo.