Drugs. 2020 Feb;80(2):209-215. doi: 10.1007/s40265-020-01262-7.
Voxelotor (Oxbryta™) is a haemoglobin S polymerization inhibitor that has been developed for the treatment of sickle cell disease. In November 2019, voxelotor received its first global approval in the USA for the treatment of sickle cell disease in adults and paediatric patients aged ≥ 12 years. The drug was granted accelerated approval based on the results of the phase III HOPE trial. Phase III clinical development of voxelotor for sickle cell disease is ongoing worldwide. Voxelotor also has Orphan Drug designation and Priority Medicine status in Europe for the treatment of sickle cell disease. This article summarizes the milestones in the development of voxelotor leading to this first approval as a disease-modifying agent for sickle cell disease.