1.1 Lanadelumab is recommended as an option for preventing recurrent attacks of hereditary angioedema in people aged 12 and older, only if:
they are eligible for preventive C1-esterase inhibitor (C1-INH) treatment in line with NHS England's commissioning policy, that is, they are having 2 or more clinically significant attacks (as defined in the policy) per week over 8 weeks despite oral preventive therapy, or oral therapy is contraindicated or not tolerated
the lowest dosing frequency of lanadelumab is used in line with the summary of product characteristics, that is, when the condition is in a stable, attack-free phase (see section 2) and
the company provides lanadelumab according to the commercial arrangement.
1.2 This recommendation is not intended to affect treatment with lanadelumab 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 or the young person's parents or carers.
Why the committee made these recommendations
People with hereditary angioedema have attacks that cause severe swelling of various parts of the body. Despite long-term oral preventive therapy (such as attenuated androgens) and C1-INH treatments, some people still have frequent severe attacks.
Lanadelumab's marketing authorisation is broad and covers prevention of recurrent attacks of hereditary angioedema. But there is no trial evidence comparing lanadelumab with long-term oral preventive therapy so it cannot be used instead of this therapy. Therefore the company wants lanadelumab to be used only for people who are eligible for long-term preventive C1-INH treatments in line with NHS England's commissioning policy. So C1-INH treatments are the most appropriate comparator for the company's proposed positioning.
Evidence from a randomised controlled trial suggests that people having lanadelumab have fewer hereditary angioedema attacks than with placebo. There are data indirectly comparing lanadelumab with C1-INHs.
Lanadelumab does not meet NICE's criteria to be considered a life-extending treatment at the end of life. In line with its summary of product characteristics, a lower dosing frequency of lanadelumab (once every 4 weeks) can be used if the condition is in a stable attack-free phase. But there is no clinical trial evidence on switching to this lower dosing frequency and the proportion of patients assumed to switch has a large impact on the cost-effectiveness estimates. Although all cost-effectiveness estimates for lanadelumab compared with C1-INHs are uncertain, most are within the range NICE normally considers an acceptable use of NHS resources. Therefore, lanadelumab is recommended only for people who are eligible for long-term preventive C1-INH treatments in line with NHS England's commissioning policy. The lowest dosing frequency of lanadelumab should be used in line with the summary of product characteristics, when the condition is in a stable attack-free phase.