A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults with Hereditary Angioedema

Patients with hereditary angioedema (HAE) will take a daily 40 mg tablet of deucrictibant for 130 weeks to see if it safely prevents the sudden, painful swelling attacks that characterise the disease. HAE is a rare genetic condition where a faulty protein pathway allows bradykinin—a molecule that makes blood vessels leaky—to build up unchecked. This causes unpredictable attacks of swelling in the hands, feet, gut, or airway, which can be life-threatening if the throat closes. Current preventive treatments often require injections or infusions, and some patients still have breakthrough attacks. This study tests whether a once-daily oral pill, deucrictibant, which blocks the bradykinin B2 receptor, can provide a simpler, long-term prophylactic option. If the drug proves safe and effective over 2.5 years, it could give HAE patients a convenient, needle-free way to dramatically reduce attack frequency. That would mean fewer emergency hospital visits, less disruption to work and daily life, and lower risk of fatal airway swelling. The trial runs across 80–90 global sites, with 16 visits (some remote), and tracks attacks via daily electronic diaries. Success would shift HAE prophylaxis from injectable biologics to an oral tablet—a practical upgrade for a condition that currently demands constant vigilance.