Casgevy

Casgevy is a medicine used to treat blood disorders known as beta thalassaemia and sickle cell disease in patients 12 years and older. For beta thalassaemia, Casgevy is used in patients who require regular blood transfusions. Patients with this condition do not make enough haemoglobin, the protein in red blood cells that carries oxygen around the body. As a result, these patients have low red blood cell levels and need frequent blood transfusions. For sickle cell disease, Casgevy is used in patients with severe disease and recurrent painful crises. Patients with this condition have an abnormal form of haemoglobin that makes red blood cells become rigid and sticky and change from being disc-shaped to being crescent-shaped (like a sickle). These cells can block blood vessels, causing painful crises that affect the chest, abdomen (belly) and other parts of the body. Beta thalassaemia and sickle cell disease are rare, and Casgevy was designated an ‘orphan medicine’ (a medicine used in rare diseases). Further information on the orphan designations can be found on the European Medicine’s Agency website ( [beta thalassaemia](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2210): 17 October 2019; [sickle cell disease](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2242): 9 January 2020) Casgevy contains as its active substance genetically modified stem cells (cells that can develop into blood cells) taken from the patient’s own blood. It is used when stem cell treatments are appropriate and there are no suitable stem cell donors available.