Berotralstat Tolerability and Effectiveness in Hereditary Angioedema: Berolife Study Results.

Authors

Delphine Gobert, David Launay, Isabelle Boccon-Gibod, Melisande Bourgoin-Heck, Cyrille Hoarau, Yann Ollivier, Hervé Maillard, Fabien Pontille, Nicolas Ozanne, Magali Aubineau, Claire de Moreuil, Anne Gerber, Fabien Pelletier, Pierre-Yves Jeandel, Aurélie Du-Thanh, Marie-Caroline Dalmas, Marie-Caroline Taquet, Jean Schmidt, Amélie Servettaz, Amandine Perier, Enzo Cohen, Laure Carriat, Nicolas Lemaire, Mona Villedieu, Jean-Charles Crave, Olivier Fain, Laurence Bouillet

Published in

Clinical and translational allergy. Volume 16. Issue 8. Pages e70193.

Abstract

Berotralstat is a first-line once-daily oral prophylactic treatment for hereditary angioedema (HAE). Berolife was designed to evaluate the tolerability and effectiveness of berotralstat in real-world conditions.
Berolife is an open-label, multicenter, observational study conducted in France from September 2021 to January 2024. The primary objective was to assess tolerability, and secondary objectives included characterization of the treated population and assessment of berotralstat effectiveness. HAE attack rate was assessed before and after initiation of berotralstat using the paired Wilcoxon rank-sum test.
Altogether, 80 patients were enrolled in the study (75 with HAE-C1INH type 1 or 2 and 5 with HAE-nC1INH) and received at least one dose of berotralstat. At baseline, patients had a mean (standard deviation [SD]) age of 40.0 (17.5) years, and most (67.5%) had received prior long-term prophylaxis treatment. The mean (SD) duration of berotralstat treatment was 11.5 (8.5) months. Treatment-related adverse events (AEs) occurred in 45.0% of patients, with gastrointestinal disorders being the most common (diarrhea: 13.8%, abdominal pain: 12.5%, upper abdominal pain: 7.5%). Importantly, no treatment-related serious AEs were reported. A total of 61 patients were treated with berotralstat for ≥ 6 months and evaluated for effectiveness. At 6 months of treatment, a significant reduction in monthly HAE attack rates was observed (mean [SD]: 0.62 [0.66] vs. mean [SD]: 1.25 [1.10] at baseline; p = 0.001).
Berotralstat was generally well tolerated with a tolerability profile consistent with prior clinical trials. Significant reductions in monthly HAE attack rates were observed, confirming its effectiveness in real-world settings.

PMID:
42603305
Bibliographic data and abstract were imported from PubMed on 16 Aug 2026.

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