Introduction: Hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE) is a rare yet still probably underdiagnosed clinical condition. Recurrent episodes of subcutaneous and sub-mucosal swelling may involve the skin, the gastrointestinal tract or even the upper airways, exposing the patients to the risk of death. With the aim of improving patients' quality of life, the therapeutic scenario has expanded over the years.Areas covered: The focus of the present review is lanadelumab, a fully human, κ-light-chain, monoclonal immunoglobulin G1 against plasma kallikrein, currently approved for long-term prophylaxis of C1-INH-HAE attacks in the USA and Canada and designated as an orphan drug by the European Medicines Agency.Expert opinion: Lanadelumab is able to inhibit plasma kallikrein with high selectivity and affinity. The subsequent phases of drug development and the ongoing open-label trial have proven its safety and efficacy. It overcomes some of the limitations of other drugs available for long-term prophylaxis, given the easy route of administration, the simple administration schedule and the possibility to tailor the treatment to each patient. Further studies are needed to test its efficacy also in other types of angioedema for which a central role of plasma kallikrein is envisaged.
Keywords: C1 inhibitor deficiency; efficacy; hereditary angioedema; kallikrein; lanadelumab; monoclonal immunoglobulin; prophylaxis; safety.