Vertex deploys Casgevy gene-editing platform across authorized NHS treatment hubs
The Medicines and Healthcare products Regulatory Agency (MHRA) approved the expanded indication, lowering the minimum eligible age from 12 to five years for both conditions.
UK—Vertex Pharmaceuticals and CRISPR Therapeutics have secured UK regulatory approval to expand the use of their gene-editing therapy Casgevy (exagamglogene autotemcel) to children aged five and above with severe sickle cell disease or transfusion-dependent β-thalassemia.
The Medicines and Healthcare Products Regulatory Agency (MHRA) approved the expanded indication, lowering the minimum eligible age from 12 to five years for both conditions.
The decision extends access to the treatment among younger patients with inherited blood disorders, potentially increasing its addressable patient population in the UK.
However, access through the National Health Service (NHS) will depend on reimbursement decisions.
The National Institute for Health and Care Excellence (NICE) recommended Casgevy for eligible patients aged 12 and above with β-thalassemia in 2024 and sickle cell disease in 2025.
Its position on treating children aged five to 11 under the expanded indication remains unclear.
Gene-editing approach targets haemoglobin production
Casgevy uses CRISPR/Cas9 gene-editing technology to modify the BCL11A gene in a patient’s blood stem cells.
This modification increases foetal haemoglobin (HbF) production, an oxygen-carrying protein normally produced before birth.
In patients with sickle cell disease, higher HbF levels help prevent red blood cells from adopting the characteristic sickle shape, reducing the risk of painful crises and other complications.
In β-thalassemia, increased foetal haemoglobin production helps improve red blood cell function and reduce the need for regular blood transfusions.
The therapy received its first US approval from the Food and Drug Administration (FDA) in 2023 for sickle cell disease, followed by a second approval in 2024 for transfusion-dependent β-thalassemia.
More recently, the FDA expanded Casgevy’s eligibility to patients aged two and above.
Vertex reported that the agency granted the approval 53 days after receiving the application, extending the treatment’s potential reach among younger patients.
Commenting on the UK decision, MHRA Executive Director of Operations Julian Beach said severe sickle cell disease and β-thalassemia are lifelong conditions that can be fatal.
He noted that the expanded indication followed an assessment of the available evidence on Casgevy’s safety and efficacy.
Commercial outlook and market access
The UK approval adds to Casgevy’s commercial prospects as Vertex and CRISPR seek to expand the use of gene-editing medicines.
Vertex reported that Casgevy revenue increased by 78% quarter-on-quarter and 151% year-on-year in the second quarter of 2026.
Analysts at GlobalData, the parent company of Pharmaceutical Technology, forecast that Casgevy could generate US$1.6 billion in annual sales by 2032.
The expansion into younger age groups could support demand, although treatment costs, specialist infrastructure, reimbursement decisions, and the complexity of administering personalized cell therapies remain important factors influencing adoption.
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