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Hereditary angioedema (HAE) is an autosomal dominant disorder in which attenuated levels or function of C1 inhibitor leads to unchecked generation of plasma kallikrein and resultant cleavage of high-molecular-weight kininogen and bradykinin production. This excess bradykinin can lead to disabling and life-threatening swelling of the extremities, face, gut, and airway. Lanadelumab, a monoclonal antibody inhibitor of kallikrein, might prevent HAE attacks.
In an industry-sponsored, phase 1B study, 28 adults with recent HAE attacks were randomized (in a 2:1 ratio) to two doses of subcutaneous lanadelumab or placebo 2 weeks apart; participants were followed for 50 days. Patients in the treatment group had significantly fewer attacks than did plac…