The CRISPR-based gene therapy Casgevy (exagamglogene autotemcel) has been made available to patients with sickle cell disease in England, following positive guidance issued by the National Institute for Health and Care Excellence (NICE).
NICE released their final draft guidance today (31 January), recommending Casgevy coverage on the National Health Service (NHS). The therapy was co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
The institute said patients aged 12 and over with severe sickle cell disease, recurrent vaso-occlusive crises, and a certain genotype would qualify for treatment.
Casgevy is a one-time therapy that uses CRISPR/Cas9 gene-editing technology to modify a patient’s haematopoietic stem cells. The process targets the BBCL11A gene to increase foetal haemoglobin production, helping red blood cells function more effectively and reducing complications associated with sickle cell disease.
Unlike donor stem cell transplants – the only curative treatment currently available – Casgevy does not carry the same risk of rejection. However, it is only recommended for patients who are eligible for a transplant but lack a suitable donor.
The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by approval for transfusion-dependent beta-thalassemia (TDT) in January 2024. NHS England reached an earlier reimbursement agreement for Casgevy in TDT in August 2024.
NICE previously declined to recommend Casgevy for NHS use in March 2024, citing insufficient evidence to justify its high cost. The therapy has a list price of £1.65m, and a commercial agreement was necessary to determine its affordability within the NHS. The confidential pricing agreement now allows Casgevy to be made available under a managed-access scheme, meaning further data will be collected as patients undergo treatment.
NHS England estimates that approximately 50 sickle cell disease patients per year will initially receive Casgevy, doubling the current number of patients who undergo stem cell transplants.
Patient representatives and advocacy groups have welcomed the agreement. Funmi Dasaglu, a sickle cell disease patient nominated by the Anthony Nolan charity to provide input to NICE, described the therapy as a transformative option. She highlighted the lack of effective treatments and the potential for Casgevy to improve patients’ quality of life.
Sickle Cell Society CEO John James stated that the approval was the result of extensive campaigning and represents a major breakthrough for patients.
NICE acknowledged that sickle cell disease disproportionately affects people from African, Caribbean, Middle Eastern, and South Asian backgrounds and stated that it considered these health inequalities when assessing Casgevy’s cost-effectiveness. The decision to approve the therapy was made with an understanding that additional evidence on long-term efficacy would be gathered over time.
Data collection through the managed-access agreement is expected to address uncertainties, particularly regarding the durability of the treatment’s benefits.
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