Bongkarn Thanyakij / Alamy Stock Photo
The Medicines and Healthcare products Regulatory Agency (MHRA) has approved a gene-editing treatment for severe sickle-cell disease and transfusion-dependent β-thalassemia for patients aged five years and older.
Casgevy (exagamglogene autotemcel; Vertex) had previously been approved by the MHRA in November 2023 for patients with sickle-cell disease or transfusion-dependent β-thalassemia, who are aged 12 years and over.
This was followed by National Institute for Health and Care Excellence (NICE) recommendations in September 2024 for eligible patients with transfusion-dependent β-thalassemia and in February 2025 for eligible patients with sickle cell disease.
On 30 October 2026, the MHRA said: “The latest approval extends its use to younger children who meet the eligibility criteria.”
On the same day, NICE published a ‘Managed access agreement‘, which confirmed that children aged 5–11 years are now eligible to receive the treatment.
“After considering the evidence submitted by the manufacturer for this population, NICE concluded that it could update the recommendations in TA1044 to include children aged 5 to 11 years without a separate technology appraisal,” the agreement said.
“NICE considered that extending the recommendation to this younger population would not materially affect the cost-effectiveness estimates or substantially change the key uncertainties identified at the original appraisal for the 12+ population.”
Julian Beach, executive director of operations at the MHRA, commented: “Severe sickle cell disease and β-thalassemia are serious, lifelong conditions that in some cases can be fatal.
“This approval means this innovative gene-editing treatment can now be used in eligible children from the age of five, extending this treatment option to younger patients. It is based on careful assessment of the available evidence on the treatment’s safety, quality and effectiveness.”
A spokesperson for NICE told The Pharmaceutical Journal: “The timing of this update has been aligned with approval of the licence extension. A streamlined process was used to consider the new evidence for this younger age group.”
On 29 October 2026, Casgevy’s manufacturer Vertex said: “[A] reimbursement agreement with NHS England, and NICE issuing updated guidance, means that eligible patients can now access the treatment in England.”
Vertex added that it was “working closely with the national health authorities in Wales, Scotland and Northern Ireland to extend access for eligible patients as quickly as possible”.
Casgevy was the first gene-editing CRISPR therapy to be recommended by NICE.
In a press release published on 23 October 2026, Manchester University NHS Foundation Trust highlighted that a patient — who according to the trust was the first patient in the UK to receive the drug for sickle cell disease — had completed her treatment. Aisha, aged 15 years, from Cheadle, Manchester, was given the treatment following NICE approval in 2025, it added.
Manchester University NHS Foundation Trust noted: “[Aisha] has now rung the bell to mark the end of her treatment. Whilst she is still receiving care from the hospital, for help with her ongoing muscle pains which were caused by damage through sickle cell, she no longer has symptoms and does not have crises.”
