Vertex Pharmaceuticals has secured a reimbursement agreement with National Health Service (NHS) England for Casgevy (exagamglogene autotemcel) in sickle cell disease (SCD).
Eligible SCD patients in England can now access this CRISPR/Cas9 gene-edited therapy.
This one-time gene-edited cell therapy comes at a price tag of £1.65m.
The reimbursement agreement follows positive guidance from the National Institute for Health and Care Excellence (NICE), recommending Casgevy for NHS use.
According to NHS, researchers found a ‘functional cure’ in 96.6% of exa-cel trial participants. All patients avoided hospitalisation for a year, with nearly 98% remaining hospitalisation-free after 3.5 years.
The latest reimbursement follows the Casgevy agreement for transfusion-dependent beta thalassemia (TDT) patients announced in August 2024.
In November 2023, Vertex Pharmaceuticals and CRISPR Therapeutics secured conditional marketing authorisation for CASGEVY in the UK to treat SCD and TDT.
NHS chief executive Amanda Pritchard said: “This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative, it could enable patients to live free from the fear of sickle cell crises hanging over them.
“It is just the latest in a series of revolutionary gene therapies NHS England has secured for patients, and we are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.”
The gene-edited cell therapy will be offered at specialist NHS centres in London, Manchester, and Birmingham.
It will be available under a managed access scheme for individuals aged 12 and over with certain severe SCD types.
The treatment involves collecting stem cells, editing them in a lab using CRISPR technology to produce non-sickling red blood cells, and infusing the edited cells back into the patient.
Administering the therapy requires expertise in stem cell transplantation and hemoglobinopathy management. US-based Vertex Pharmaceuticals said that it is working with hospitals across England to create a network of authorised treatment centres (ATCs).
Vertex international senior vice president Ludovic Fenaux said: “We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with CASGEVY, recognising the value a one-time treatment can provide to patients, their families and the healthcare system.”
The biotechnology company is also advancing clinical programmes in neuropathic pain, IgA nephropathy, kidney diseases, type 1 diabetes, and myotonic dystrophy type 1.
