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Crizanlizumab for preventing sickle cell crises in sickle cell disease

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1 Recommendations

1.1 Crizanlizumab is not recommended, within its marketing authorisation, for preventing recurrent sickle cell crises (vaso-occlusive crises) in people aged 16 or over with sickle cell disease.

This recommendation is not intended to affect treatment with crizanlizumab that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Why the committee made these recommendations

Treatments to prevent sickle cell crises include hydroxycarbamide (also known as hydroxyurea), which is taken as a tablet, or regular blood transfusions. Crizanlizumab is a treatment injected into the vein (intravenous, or IV) that people aged 16 or over can take on its own or alongside hydroxycarbamide.

The clinical evidence suggests that people taking crizanlizumab have fewer sickle cell crises in a year than if they do not have any treatment. However, these results are not certain because the trial was short and included only a small number of people on the licensed dose of the drug.

There is also uncertainty about the cost-effectiveness estimates. The most likely cost-effectiveness estimate is substantially higher than what NICE normally considers a cost-effective use of NHS resources. So, crizanlizumab is not recommended for use in the NHS.