RegulatoryOfficial

MHRA extends Casgevy gene-editing authorisation to children aged five and over

🇬🇧 MHRA · Fri 30 Oct 2026

The UK regulator has widened Casgevy's licence from patients aged 12 and over to eligible younger children with severe sickle-cell disease or transfusion-dependent β-thalassaemia.

The Medicines and Healthcare products Regulatory Agency (MHRA) has extended the UK marketing authorisation for Casgevy (exagamglogene autotemcel) to children aged five and over. The therapy is indicated for severe sickle-cell disease and transfusion-dependent β-thalassaemia in those who meet the eligibility criteria. It was previously authorised only for patients aged 12 and over.

The extension was granted on 29 October 2026 to Vertex Pharmaceuticals (Europe) Ltd, and the MHRA announced it the following day. The agency says the Summary of Product Characteristics and Patient Information leaflets will appear on the MHRA Products website within seven days of approval.

Casgevy edits a faulty gene in a patient's own bone marrow stem cells so that the body produces functioning haemoglobin. The cells are collected, edited in a laboratory and infused back after conditioning treatment. The MHRA notes that patients may need to stay in hospital afterwards while the treated cells establish themselves in the bone marrow.

The agency lists nausea, fatigue, fever and an increased risk of infection among the side effects associated with the treatment. Julian Beach, MHRA Executive Director Operations, said the decision rests on careful assessment of the evidence on safety, quality and effectiveness, and that the agency will keep monitoring the safety and effectiveness of Casgevy.

The MHRA says about 15,000 people in the UK have sickle cell disorder. It adds that patients with β-thalassaemia often need a blood transfusion every three to five weeks, as well as injections and medicines throughout their lives.

What it means for your team

  • Regulatory teams should check the updated SmPC and patient leaflet once published, expected within seven days of approval, for the paediatric indication wording.
  • Pharmacovigilance teams should note that the MHRA has said it will keep monitoring safety and effectiveness, so the younger patient group falls within ongoing post-authorisation surveillance.
  • Treatment centres and market-access teams should expect a wider eligible population than the 12-and-over group covered until now; the source gives no detail on funding or access arrangements.

Official source: MHRA ↗

Article written by VigiEye from the official source (MHRA). AI-assisted; check the source before acting.

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