RAPID AND SUSTAINED REDUCTIONS IN HEREDITARY ANGIOEDEMA ATTACK RATES WITH LONG-TERM BEROTRALSTAT: REAL-WORLD OUTCOMES

Abstract

<h3>Introduction</h3> Berotralstat, an oral once-daily prophylactic treatment for hereditary angioedema (HAE), has demonstrated sustained reduction in HAE attack rates and improvements in patient-reported quality of life in clinical trials. Here we present the preliminary efficacy data of patients in the United States receiving berotralstat in the real-world clinical setting. <h3>Methods</h3> This analysis is data collected through the sole-source pharmacy and included patients with confirmed HAE Type I/II based on laboratory tests and actively received berotralstat 110 or 150mg from 12/16/2020 to 5/20/22 for >270 days. Baseline attack rates were reported for the 90 days prior to berotralstat initiation. While on therapy, median attacks/month (25^th,75^th percentile) were calculated over each 90-day period by averaging each patient's monthly reported attack rate (1 month=∼28 days). Some patients did not report attack rates at each refill. <h3>Results</h3> In the patients who actively received berotralstat for >270 days (n=128) the median baseline attack rate was 1.7 attacks/month. A rapid reduction in median monthly attack rates was observed after starting berotralstat. During the first 90 days on therapy (days 1-90) the median monthly attack rate decreased to 0.3 (0,1.50) attacks/month; 0.5 (0,1.00) attacks/month from days 91-180; 0.3 (0,1.25) attacks/month from days 181-270; and 0.3 (0,1.25) attacks/month from days 271-360. The most common adverse events reported were consistent with clinical trials. <h3>Conclusions</h3> This data shows that berotralstat provided a rapid and sustained reduction in HAE attack rates within the first months of starting therapy making oral berotralstat an effective treatment option for patients with HAE.