1.1 Berotralstat is recommended as an option for preventing recurrent attacks of hereditary angioedema in people 12 years and older, only if:
it is stopped if the number of attacks per month does not reduce by at least 50% after 3 months.
It is only recommended if the company provides berotralstat according to the commercial arrangement.
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
There are not many effective treatments available for preventing recurrent attacks of hereditary angioedema. Clinical trial evidence suggests that berotralstat is effective at reducing the number of attacks per month compared with placebo.
Despite some uncertainty in the clinical evidence, berotralstat is considered cost effective for people who have at least 2 attacks per month, and if they stop having berotralstat if it has not reduced attacks enough after 3 months. So, it is recommended for these people.