Sickle cell gene therapy Casgevy finally cleared for NHS use

Vertex Pharma's £1.65 million ($2.05 million) gene-editing therapy for sickle cell disease (SCD), Casgevy, has been given the green light for use by the NHS in England under a managed access scheme and with a confidential discount.

The CRISPR-based therapy will be an option for SCD patients aged 12 years and over who have at least two vaso-occlusive crises (VOCs) – the excruciatingly painful attacks that occur when red blood cells become misshapen and block blood vessels – per year.

Treatment with Casgevy (exagamglogene autotemcel or exa-cel) involves collecting stem cells from a patient, editing them to produce blood cells that do not form the characteristic sickle shape seen in SCD, and reinfusing them into the body.

It will be an option for people who might otherwise be treated using a bone marrow transplant, but have no suitable donor available. Moreover, as it is based on the patient's own cells, there is no chance of the transplant being rejected by the recipient's immune system.

The decision means that SCD patients in England will have access to the drug, along with transfusion-dependent beta-thalassaemia (TDT) patients for whom the use of the therapy was recommended last August. Casgevy was approved in the UK for SCD in 2023 and for TDT last year.

The final draft guidance comes after NICE's appraisal committee heard from a patient expert who described how exa-cel had made them "healthier, fitter, and stronger than at any point in my life before," according to the health technology assessment (HTA) agency.

It is estimated that around 15,000 people in England live with SCD, which mainly affects people of African, Caribbean, Middle Eastern, or South Asian heritage.

John James, chief executive of the Sickle Cell Society, said: "We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. The significance of this milestone for the sickle cell community cannot be understated."

The patient organisation has been lobbying hard to extend the use of Casgevy to include SCD patients since NICE declined to recommend it for this indication in March 2024.

"While today's news is incredible, we remain acutely aware that not everyone with sickle cell will be eligible for the potentially life-changing benefits of Casgevy," said James. "Furthermore, this new therapy joins a disappointingly limited range of treatments currently available for this complex and challenging condition."

Vertex said it is working with experienced hospitals throughout England to establish a network of treatment centres that can deliver Casgevy.