Exagamglogene autotemcel for treating severe sickle cell disease in people 12 years and over

  • Technology appraisal guidance
  • TA1044
  • Published: 

Information for the public

Exagamglogene autotemcel (exa‑cel; Casgevy) is available during the managed access period as a possible treatment for sickle cell disease in people 12 years and over. It is for:

  • people who have:
    • recurrent episodes of severe pain, called vaso-occlusive crises (defined in section 3.4 of the guidance) and
    • a βS/βS, βS/β+ or βS/β0 genotype, and
  • when a haematopoietic stem cell transplant (a blood and marrow transplant) is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available.

It is only recommended for people who have had at least 2 vaso-occlusive crises per year during the 2 previous years.

More evidence on exa‑cel is being collected. After this, NICE will decide whether to recommend it for routine use on the NHS and update the guidance. It will be available with managed access until then.

If you are not eligible for exa-cel but you have already begun the process of having it, you should be able to complete the treatment unless you and your healthcare professional decide to stop.

Is this treatment right for me?

Your healthcare professionals should give you clear information, talk with you about your options and listen carefully to your views and concerns. Your family can be involved too, if you wish. See our webpage on making decisions about your care.

Questions to think about

  • How well does it work compared with other treatments?
  • What are the risks or side effects? How likely are they?
  • How will the treatment affect my day-to-day life?
  • What happens if the treatment does not work?
  • How long does the treatment take?
  • How many hospital visits will I need to make?
  • What happens if I do not want to have this treatment? Are there other treatments available?

Information and support

The NHS webpage on sickle cell disease may be a good place to find out more.

These organisations can give you advice and support:

You can also get support from your local Healthwatch.

NICE is not responsible for the quality or accuracy of any information or advice provided by these organisations.

ISBN: 978-1-4731-6872-5

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