gene-editing treatment
Published on 30 Oct 2026 · source: MHRA — Medicines and Healthcare products Regulatory Agency
What this means
The MHRA has announced the approval of a medicine or a new indication in the UK. Approval does not mean the product is already available on the NHS: NICE appraisal and commissioning may follow.
Summary
Children aged five and over with severe sickle-cell disease and transfusion-dependent β-thalassemia, can now be treated with Casgevy (exagamglogene autotemcel), following an extension of its UK authorisation by the MHRA.