The treatment is recommended only for people who have at least
2 attacks per month and should be stopped if the number of
attacks per month does not reduce by at least 50% after 3
months of use.
Hereditary angioedema is a rare genetic disorder affecting
approximately 1 person per 50,000 to 100,000. It is a relapsing
condition that causes unpredictable and recurrent attacks of
swelling in the mouth, gut or airway. It often leads to
difficulty breathing and severe pain, and can cause swelling in
multiple places in the body at once.
The aim of preventive treatment is to reduce the rate and
severity of attacks and allow people to live an attack-free
life. Currently there are few effective preventative treatment
options for patients and access is limited, with some
treatments only available to people who have attacks 2 or more
times per week.
The new draft guidance means that close to one third of
patients with the condition in England will be eligible for
berotralstat, the first oral treatment for this condition to be
made available on the NHS.
Clinical trial evidence on the effectiveness of berotralstat
suggests that it reduces the frequency of swelling attacks,
though the effect on the severity of these attacks is
uncertain.
Berotralstat was previously available through the Early Access
to Medicines Scheme. Although earlier draft guidance from NICE
did not recommend the treatment for routine use, the committee
has since seen more data and we can now recommend it for use in
the NHS.
Read the draft guidance.
