Three young people chatting and laughing in an outside dining area at night

Our independent appraisal committee has approved the use of gene editing therapy exagamglogene autotemcel (exa-cel) for use in the NHS in England, providing a potential cure for some people with severe sickle cell disease (SCD).

It will be made available under a managed access scheme for some people 12 years and over with certain types of severe SCD.

Treatment with exa-cel (also called Casgevy and made by Vertex) involves collecting the person’s stem cells. These are then edited in a lab to produce non-sickling red blood cells. The edited cells are then infused back into the person.

As exa-cel involves people receiving their own edited cells, they have no risk of their body rejecting them.

The committee heard from a patient expert who described how the distressing lifelong disease had affected them and that treatment with exa-cel had made them "healthier, fitter and stronger than at any point in my life before".

Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing NICE’s aim of reducing health inequalities associated with the condition and getting the best care to patients fast.

Funmi Dasaolu, who has sickle cell disease and was nominated by charity Anthony Nolan to tell her story to the NICE committee, said: "The approval of exa-cel today marks a significant shift in the treatment landscape of sickle cell disease in the UK. It is the beginning of re-addressing the inequalities in care experienced by so many with the condition. It provides much needed hope and will undoubtedly radically transform the lives of those living with this genetic blood disorder.”

There are very few treatments to stop symptoms of SCD and those that are available often have intolerable side effects. Currently the only curative treatment for people with SCD is a donor stem cell transplant. Exa-cel will now be an option when a stem cell transplant is suitable but no matched donor can be found.  

The committee took into account exa-cel’s potential impact on health inequalities (SCD is more common in people from African, Caribbean, Middle Eastern or South Asian family backgrounds by allowing more uncertainty in the evidence and a higher cost-effectiveness estimate than NICE normally considers to be value for money for the NHS.

More data will be collected while patients receive the treatment on the NHS before NICE evaluates the medicine again. Collecting more data through a managed access agreement may resolve some uncertainty in the evidence.

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