Attack rate reductions following berotralstat initiation among US patients with hereditary angioedema in the real-world

Abstract Background Hereditary angioedema (HAE) causes recurring swelling attacks, leading to substantial disease burden. This real-world, retrospective study aimed to evaluate HAE attack rates before and after berotralstat initiation stratified by patients’ baseline attack frequency. Methods Specialty Pharmacy data from Optime Care, Inc. (12/2020–01/2024), the sole berotralstat dispenser in the United States, were analyzed. Eligible patients had ≥ 2 berotralstat dispensings (first = index) and ≥ 1 self-assessment of attacks at baseline (90-days pre-index) and follow-up (first-to-last dispensing). Patients were classified by HAE type (based on laboratory measurements) and baseline attacks (≥ 5, 2–4, 1, and 0 attacks/month). Follow-up attack rates were compared with baseline using mean differences, confidence intervals, and P-values. Among those with 0 baseline attacks/month, proportions with 0 follow-up attacks/month were assessed. Results Of 390 eligible patients with HAE with C1 esterase inhibitor (C1INH) deficiency (HAE-C1INH) and 311 with HAE with normal C1INH (HAE-nC1INH), most were female (64.1% and 77.5%) with mean ages of 39.3 and 48.1 years, respectively. Mean attack rates decreased from 2.50 to 0.79 attacks/month (HAE-C1INH) and from 4.59 to 1.68 attacks/month (HAE-nC1INH) at 12-months of berotralstat treatment (both P < 0 .001), with sustained reductions at 18-months. Patients with ≥ 1 baseline attack/month experienced significantly lower attack rates after berotralstat initiation. Among patients with 0 baseline attacks/month, most also maintained 0 attacks/month in each follow-up interval (HAE-C1INH: 70–85%; HAE-nC1INH: 61–81%). Conclusion Berotralstat was associated with significant and sustained reductions in attack rates among patients with HAE, regardless of baseline attack rate. Patients with 0 baseline attacks/month maintained low attack rates following berotralstat initiation.