Oxbryta

Oxbryta is a medicine used to treat haemolytic anaemia (excess breakdown of red blood cells) in patients aged 12 years and older who have sickle cell disease. Oxbryta can be given on its own or together with another medicine for sickle cell disease called hydroxycarbamide. Sickle cell disease is a genetic disease where individuals produce an abnormal form of haemoglobin (the protein in red blood cells that carries oxygen). The red blood cells become rigid and sticky, and change from being disc-shaped to being crescent-shaped (like a sickle). Sickle cell disease is rare, and Oxbryta was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 18 November 2016. Further information on the orphan designation can be found here: [ema.europa.eu/medicines/human/orphan-designations/eu-3-16-1769](/en/medicines/human/orphan-designations/eu-3-16-1769). Oxbryta contains the active substance voxelotor.