Berotralstat Associated With Significant Reductions in Healthcare Utilization in HAE With Normal C1-Inhibitor

Real-world data suggest that initiation of berotralstat, an oral long-term prophylaxis for hereditary angioedema (HAE), is associated with meaningful reductions in angioedema-related healthcare resource utilization among patients with HAE with normal C1-inhibitor (HAE-nC1INH).

“Angioedema-related healthcare resource utilization was evaluated pre- and post-initiation of berotralstat, the only targeted oral long-term prophylaxis for HAE, among patients with hereditary angioedema with normal C1-inhibitor (HAE-nC1INH),” the investigators stated in an abstract presented at the American Academy of Allergy, Asthma, and Immunology Annual Meeting in Philadelphia February 27-March 2.

This retrospective analysis used tokenized Optime Care Specialty Pharmacy data linked with the Komodo Healthcare Map between December 3, 2020, and June 30, 2024. Eligible patients had at least 2 berotralstat dispensings, 6 months of pre-index insurance enrollment, and confirmed HAE-nC1INH based on physician diagnosis and normal C1-inhibitor and C4 levels. Optime Care, the sole US dispenser of berotralstat, also collected patient-reported attack frequency, allowing stratification by baseline attack rates (≥5, 2–4, 1, or 0 attacks per month in the 90 days before treatment initiation).

Among 178 eligible patients (mean age, 47.1 years; 79.2% female), mean rates of angioedema-related outpatient or emergency department (ED) visits and hospitalizations before berotralstat initiation were 6.6 and 1.1 per patient-year, respectively.

After starting berotralstat, angioedema-related outpatient/ED visits decreased by 51% and hospitalizations by 36% (both P<.05). Reductions in outpatient/ED visits ranged from 44% to 64% across baseline attack-rate subgroups (P<.05), while hospitalizations declined by 17% to 56%, although subgroup differences were not statistically significant.

“Following the initiation of berotralstat, patients with HAE-nC1INH experienced significant reductions in angioedema-related outpatient/ED visits and hospitalizations regardless of their baseline attack rate,” the authors concluded.

Reference
Zuraw B, Yee T, Laliberte F, Spencer C, Nestler-Parr S, Gillard P, Christiansen S. Impact of berotralstat on healthcare resource utilization in patients with hereditary angioedema with normal C1-inhibitor. Presented at: American Academy of Allergy, Asthma, and Immunology Annual Meeting. February 27-March 2, 2026; Philadelphia, Pennsylvania.