Berotralstat effectiveness and safety in patients with hereditary angioedema with normal C1 inhibitor
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Authors
Buckland, Matthew S.
Boccon-Gibod, Isabelle
De Moreuil, Claire
Sanges, Sébastien
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Issue Date
2026
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Article
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BACKGROUND: Hereditary angioedema (HAE) with normal C1 inhibitor (nC1-INH) is the least common endotype of HAE, a rare disorder with localized, intermittent attacks of soft tissue swelling. Prophylactic treatments are available across HAE endotypes, including berotralstat, a once-daily oral inhibitor of plasma kallikrein. OBJECTIVE: This European multicenter case series aimed to report treatment-related outcomes with berotralstat in patients with HAE-nC1-INH. METHODS: A retrospective case series analysis including observational data from patients in United Kingdom and French centers was performed. Patients were included if they had a genetic assay demonstrating a known HAE-nC1-INH-associated variant; or had a family history of angioedema and normal C1-INH level and function, no response to antihistamine treatment, met predefined diagnostic criteria for HAE-nC1-INH, and had been prescribed berotralstat 150 mg daily for at least 6 months. Data were collected from the patients' records using a standardized form. RESULTS: Four female and 2 male patients with HAE-nC1-INH were included from 4 centers in France and the United Kingdom. Duration of berotralstat treatment at analysis ranged from 6 to 23 months. Five patients showed a response to berotralstat, observed as a 29-100% reduction in attack rates. Three patients experienced a reduction in attack severity from moderate to minor or mild. One patient reported adverse events during berotralstat initiation: nausea and diarrhea. CONCLUSION: Long-term prophylactic berotralstat was effective in reducing HAE attacks for 5 of 6 patients with HAE-nC1-INH. No significant safety signals were noted.
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The journal of allergy and clinical immunology.Global
Volume
5
Issue
1