Long-Term Safety and Efficacy of Voxelotor for Patients with Sickle Cell Disease: Results from an Open-Label Extension of the Phase 3 HOPE Trial

Background: Sickle cell disease (SCD) is a lifelong, inherited blood disorder, resulting from a mutation in the hemoglobin (Hb) subunit β gene, that leads to sickle hemoglobin (HbS) formation. Polymerization of HbS causes red blood cell sickling and damage, leading to hemolysis, chronic anemia, and vaso-occlusive crises (VOCs). Patients with SCD are at higher risk of end-organ damage, increased morbidity, and early mortality as a result of low Hb and increased hemolysis.