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.

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.

Around 1,700 people could be eligible for the one-time treatment, which is seen as a less risky alternative to a donor stem cell transplant

A groundbreaking £1.65million gene therapy treatment offering hope for sickle cell disease patients has been approved for use on the NHS in England. The one-time treatment, called Casgevy (exa-cel), has shown remarkable success in clinical trials,

The National Institute for Health and Care Excellence (NICE) has approved Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel) gene therapy to treat a severe form of sickle cell disease (SCD).

Sickle cell patients share delight over approval of new treatment - Health officials have approved a £1.65 million treatment for some NHS patients.

It is believed around 1,700 people could be eligible for the treatment, while the NHS estimates around 50 people a year will receive it.

Read about exa-cel being rolled out on the NHS, providing a 'functional cure' for some patients with severe sickle cell disease.

The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts say.

Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care Excellence (NICE) said on Friday.