Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease

Aims Voxelotor (previously GBT440) is a haemoglobin (Hb) modulator that increases Hb‐oxygen affinity, thereby reducing Hb polymerization and sickling of red blood cells (RBCs), being developed as a once‐daily oral drug to treat sickle cell disease (SCD). This first‐in‐human study evaluated the safety, tolerability, pharmacokinetics and pharmacodynamics of voxelotor in healthy volunteers and SCD patients.