Current drugs in early development for hereditary angioedema: potential for effective treatment.

Hereditary angioedema (HAE) through C1 inhibitor deficiency is a rare but important disease. It is characterized by recurrent episodes of angioedema, which commonly affects the skin (in the form of swelling in the extremities, face and genitals) as well as the gastrointestinal tract (abdominal pain attacks). In approximately 1% of cases of angiodema-related swelling, there is obstruction of the upper airway, which is potentially life-threatening. Therefore, HAE due to C1 inhibitor deficiency may be associated with significant morbidity and mortality. Recent research has added to our ever-increasing understanding of the pathogenesis of HAE, which has, in addition, new clinical trials with new therapeutic agents and strategies. The following editorial covers drugs currently under investigation that have the potential to be promising new therapeutic options. While some compounds show promise for the future, there are currently no oral treatments available for the treatment of acute attacks. Furthermore, some of the intravenous therapies currently available require numerous injections and do not always prevent acute attacks. Attenuated androgens also may have problematic side effects, highlighting the need for new treatment options.