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UK Extends Casgevy Authorisation to Children From Age Five

lite News Desk 2 min read

Casgevy’s UK authorisation now covers eligible patients from age five with severe sickle-cell disease or transfusion-dependent β-thalassemia, according to the Medicines and Healthcare products Regulatory Agency (MHRA). The regulator granted the marketing-authorisation extension to Vertex Pharmaceuticals (Europe) Ltd on 29 October 2026. Previously, the treatment was authorised for these disorders only from age 12; the MHRA said the extension includes younger eligible children.

Both conditions are inherited and affect haemoglobin, which carries oxygen in red blood cells, according to the MHRA. The agency describes severe sickle-cell disease as a condition that can cause episodes of intense pain, anaemia and serious infections. With β-thalassemia, severe anaemia can develop, and patients often require transfusions at intervals of three to five weeks, alongside continuing medication or injections.

The treatment, also known as exagamglogene autotemcel, uses blood-forming stem cells taken from the patient. According to the MHRA’s description, those cells undergo CRISPR/Cas9 editing outside the body before being returned by infusion. Preparation for that infusion includes conditioning treatment for the bone marrow.

European Medicines Agency (EMA) product documentation explains that the edit reduces the activity of BCL11A in cells that produce red blood cells. This increases production of fetal haemoglobin. For sickle-cell disease, the mechanism is intended to reduce sickling of red blood cells; for transfusion-dependent β-thalassemia, it addresses the imbalance in globin associated with the condition.

The EMA’s explanation concerns how Casgevy works generally. It does not describe treatment outcomes for the younger children brought within the UK authorisation by this extension.

The MHRA said hospital care may be necessary after infusion while the edited cells become established in the bone marrow. Its description of the treatment also identifies associated side effects, including fever, nausea, fatigue and a greater risk of infection.

For product information following the approval, the MHRA said it would publish revised documents covering product characteristics and information for patients on its products website. The agency gave a publication timeframe of seven days from the approval date.

* This article is based on publicly available information at the time of writing.

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