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Validation of Patient-Reported Outcomes in the On-Demand Treatment of Hereditary Angioedema Attacks Published in Clinical Reviews in Allergy & Immunology
Globenewswire· 2026-03-11 10:50
Core Insights - Pharvaris has published results from a non-interventional study assessing patient experiences during hereditary angioedema (HAE) attacks, which supports the inclusion of meaningful endpoints in clinical studies for on-demand treatment [1][2][4] Company Overview - Pharvaris is a late-stage biopharmaceutical company focused on developing oral bradykinin B2 receptor antagonists to address unmet needs in bradykinin-mediated diseases, including HAE and acquired angioedema due to C1 inhibitor deficiency [1][9] - The company is preparing marketing authorization applications for its immediate-release capsule of deucrictibant as an on-demand treatment for HAE attacks, with a pivotal Phase 3 study ongoing for the extended-release tablet [9] Study Findings - The study utilized a mixed methods approach, combining quantitative real-time electronic patient-reported outcome (PRO) assessments during HAE attacks with qualitative post-attack interviews [3][4] - Key symptoms such as skin swelling, abdominal pain, and difficulty swallowing were identified as important by participants, with even small improvements considered meaningful [4][5] - The study validated the reliability and sensitivity of the Patient Global Impression of Change (PGI-C) and Angioedema symptom Rating scale (AMRA) instruments for assessing patient experiences during HAE attacks [4][6] Clinical Implications - The findings from the study are expected to inform the design of clinical trials for on-demand treatments of HAE attacks, including the hierarchy of endpoints in the Phase 3 RAPIDe-3 trial of deucrictibant [5][6] - The study reinforces the importance of patient-reported outcomes in guiding treatment decisions and developing new therapies for HAE [2][6]