New drug for rapid, needs-based self-medication of hereditary angioedema (HAE)

In the hereditary disease hereditary angioedema (HAE), swellings occur in one or more parts of the body without a recognisable trigger. Especially if the upper respiratory tract is affected, a so-called HAE attack can be life-threatening. Active substances for on-demand medication are so far only available in the form of injections and infusions. In a phase 2 study led by the University Hospital Frankfurt and the Charité – Universitätsmedizin Berlin, it has now been shown for the first time that an orally administered active substance is effective. The therapy could replace the much more time-consuming and stressful injections.

For more information, please see the official press release and the abstract to the paper below.

Press Release Universitätsklinikum Frankfurt

Abstract 

Aygören-Pürsün E, Zanichelli A, Cohn DM, Cancian M, Hakl R, Kinaciyan T, Magerl M, Martinez-Saguer I, Stobiecki M, Farkas H, Kiani-Alikhan S, Grivcheva-Panovska V, Bernstein JA, Li HH, Longhurst HJ, Audhya PK, Smith MD, Yea CM, Maetzel A, Lee DK, Feener EP, Gower R, Lumry WR, Banerji A, Riedl MA, Maurer M. An investigational oral plasma kallikrein inhibitor for on-demand treatment of hereditary angioedema: a two-part, randomised, double-blind, placebo-controlled, crossover phase 2 trial. Lancet. 2023 Feb 11;401(10375):458-469. doi: 10.1016/S0140-6736(22)02406-0. PMID: 36774155.