Evaluating deucrictibant for the treatment of hereditary angioedema

Abstract

ABSTRACT Introduction Hereditary Angioedema (HAE) is a rare disease affecting approximately 1 in 50,000 people. It results in angioedema of the skin, abdominal tract and upper airway that results in disfiguration, abdominal pain and potential for asphyxia. Early therapies for HAE have significant drug burden due to intravenous administration (C1-inhibitor) or adverse events (androgens); however, newer therapies have reduced injection requirements, and some are oral. Over the past 6 years two oral therapies have been developed for HAE to include berotralstat for long term prophylaxis (LTP) and sebetralstat for on-demand therapy (ODT). Presently, deucrictibant, another oral HAE therapy, is under investigation and appears effective for LTP and ODT. Areas covered This manuscript summarizes the development, pharmacokinetics, pharmacodynamics, and currently available clinical evidence regarding deucrictibant in HAE management. Expert opinion Deucrictibant is a promising therapeutic agent with a well-defined mechanism of action. Published and ongoing clinical studies demonstrate favorable safety, tolerability, and efficacy profiles for both LTP and ODT, offering a convenient oral alternative that may reduce treatment burden and improve patient quality of life.