About Voxelotor in Sickle Cell Disease

Voxelotor is an oral, once-daily therapy for the treatment of sickle cell disease (SCD). A sickle hemoglobin (HbS) polymerization inhibitor, voxelotor works by increasing the affinity of hemoglobin for oxygen. This stabilizes red blood cells in an oxygenated state, preventing hemoglobin polymerization and the resultant sickling and destruction of the red blood cells.

In recognition of the critical need for new SCD treatments, the U.S. Food and Drug Administration (FDA) has granted voxelotor Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. The European Medicines Agency (EMA) has included voxelotor in its Priority Medicines (PRIME) program, and the European Commission (EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD.