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Berotralstat for preventing recurrent attacks of hereditary angioedema

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1 Recommendations

1.1 Berotralstat is not recommended, within its marketing authorisation, for preventing recurrent attacks of hereditary angioedema in people 12 years and older.

1.2 This recommendation is not intended to affect treatment with berotralstat 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. For young people, this decision should be made jointly by the clinician and the young person and the young person's parents or carers.

Why the committee made these recommendations

Hereditary angioedema causes severe swelling of various parts of the body. Treatments for preventing recurrent attacks are limited. Berotralstat is an oral treatment used to prevent recurrent attacks.

Clinical trial evidence suggests that berotralstat is effective at reducing the rate of attacks compared with placebo, but by how much is unclear. The evidence is uncertain because the trial:

  • was short

  • only included a small number of people

  • did not adequately measure if berotralstat reduces attack severity, which patients and clinical experts explained is as important as reducing the number of attacks.

Because of the clinical uncertainty the economic model is uncertain too, particularly because of the small number of people in the trial. Also, the model assumes that people would stop treatment with berotralstat if the number of attacks they have does not reduce enough, which might not be appropriate in clinical practice.

Berotralstat does not meet NICE's criteria to be considered a life-extending treatment at the end of life. Also, the most likely cost-effectiveness estimates are higher than what NICE considers a cost-effective use of NHS resources. So berotralstat is not recommended.