A related editorial highlights several new and meritorious features of this RCT for sickle cell disease, a condition for which there have been limited disease-modifying therapies. Authors’ state the haemoglobin response and reduction in haemolysis observed with this orally administered, once-daily medication with side effects that minimally affect lifestyle may make voxelotor a promising advancement in the management of sickle cell disease if approved by the FDA.