The Medicines and Healthcare products Regulatory Agency has approved an extension to the use of Casgevy (exagamglogene autotemcel), allowing children aged five and over with severe sickle cell disease and transfusion dependent beta thalassaemia to receive the gene editing treatment. The treatment was previously authorised in the United Kingdom for patients aged 12 and over. The extension was granted on October 29, 2026 to Vertex Pharmaceuticals (Europe) Ltd and announced on October 30.
Casgevy works by editing a gene in a patients own bone marrow stem cells so the body produces functioning haemoglobin. The cells are collected, edited in a laboratory and infused back after conditioning treatment. Julian Beach, MHRA Executive Director of Operations, said the approval means the treatment can now be used in eligible children from the age of five, and that the decision followed assessment of the available evidence on safety, quality and effectiveness.
The regulator lists nausea, fatigue, fever and a higher risk of infection among reported side effects, and notes that patients may need to stay in hospital after infusion. About 15,000 people in the United Kingdom have a sickle cell disorder, and patients with beta thalassaemia often need a blood transfusion every three to five weeks, according to the agency.
Two cautions belong with the headline. The MHRA notice is an authorisation decision and publishes no trial data of its own. And authorisation is not the same as funded access: for the existing 12 and over population, NHS availability followed a separate appraisal and commercial agreement well after the original 2023 approval, and no equivalent funding decision for the five to eleven group has been announced. Vertex reports third quarter results after the close on November 2, where Casgevy uptake is a stated investor focus.