Hereditary angioedema (HAE) with C1 inhibitor deficiency (C1-INH-HAE) is a rare genetic disease characterized by recurring, frequently painful swelling episodes that can disrupt quality of life and may be fatal if treatment is delayed.

Treatment options for C1-INH-HAE have grown over the last decade, with the publication of several well-designed clinical trials evaluating acute and prophylactic treatment, and subsequent approval of agents with diverse targets within the kallikrein–kinin pathway. In the USA, for instance, seven HAE-specific therapies have been approved by the US FDA since 2008.

Findings from phase I studies suggest that lanadelumab, a fully human monoclonal antibody targeting plasma kallikrein, is well tolerated and can be dosed less frequently than currently available prophylactic options. This agent was recently approved in the USA and Canada for prophylaxis to prevent attacks of HAE in patients aged ≥ 12 years.