Vichinsky E, et al. - Via a multicenter, phase 3, double-blind, randomized, placebo-controlled trial with 274 patients, researchers assessed the effectiveness and safety of two dose levels of voxelotor with placebo in patients with sickle cell disease. Participants were randomly assigned in a 1:1:1 ratio to receive a once-daily oral dose of 1500 mg of voxelotor, 900 mg of voxelotor, or placebo. A significant increase in hemoglobin levels and reduction of markers of hemolysis were noted in patients using voxelotor. Furthermore, larger reductions from baseline in the indirect bilirubin level and percentage of reticulocytes were also noted in the voxelotor (1500-mg) group vs the placebo group. Investigators determined that most adverse events were not related to the trial drug or placebo. Hence, a disease-modifying potential and the inhibition of deoxygenated sickle hemoglobin polymerization was indicated.