MHRA approves gene-editing treatment for children aged five and over with serious inherited blood disorders
What was announced
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.
Key points, in the publisher's words
- The Medicines and Healthcare products Regulatory Agency (MHRA) 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 β-thalassemia to receive the gene editing treatment.
- The treatment was previously authorised in the UK for patients 12 and over with these serious inherited blood disorders. The latest approval extends its use to younger children who meet the eligibility criteria.
- Both severe sickle cell disease and β-thalassemia are genetic conditions caused by errors in the genes for haemoglobin, which is used by red blood cells to carry oxygen around the body.
Figures in this announcement
Each line is quoted from the original. Figures are as the publisher stated them on the day.
- “Approximately 15,000 people in the UK have sickle cell disorder.”
Who and where
UK bodies of power
- Medicines and Healthcare products Regulatory Agency (executive agency) · more news
- Department of Health and Social Care (UK government department) · more news
Where this sits in the 10 levels of power
Topics, as GOV.UK files them: Marketing authorisations, variations and licensing guidance.
Receipt
- Original: https://www.gov.uk/government/news/mhra-approves-gene-editing-treatment-for-children-aged-five-and-over-with-serious-inherited-blood-disorders
- Published 2026-10-30 09:18:29 UTC
- Read by this site 2026-10-30 09:30:12 UTC from GOV.UK
- Saved copy: receipt 4830, with its SHA-256 fingerprint
Contains public sector information licensed under the Open Government Licence v3.0.
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