Casgevy was today approved by the UK's medicines watchdog for over-12s. The drug treats sickle-cell disease and transfusion-dependent β-thalassemia. Thousands of Brits suffering from two blood disorders could be given a first-of-its-kind treatment that could potentially cure them. Casgevy was today approved by the UK's medicines watchdog for all over-12s after 'rigorous' safety, quality and effectiveness checks.
The drug, which is expected to be priced around £1million per patient, treats sickle cell disease and transfusion-dependent β-thalassemia — painful, life-long conditions that, in severe cases, can be fatal. Experts say the gene-editing treatment acts as a 'functional cure' for both disorders, removing the faulty gene and relieving symptoms. However, it will only be rolled out on the NHS if it receives a further approval from the National Institute for Health and Care Excellence (Nice). Sickle cell disease patients, of which there are around 15,000 in the UK, do not make haemoglobin properly — a substance in red blood cells, which carry oxygen around the body