CONSISTENTLY LOW HEREDITARY ANGIOEDEMA ATTACK RATES OBSERVED WITH BEROTRALSTAT REGARDLESS OF PREVIOUS PROPHYLAXIS: REAL-WORLD OUTCOMES

Abstract

<h3>Introduction</h3> Clinical trial data showed patients who switched to oral berotralstat monotherapy reported consistently low hereditary angioedema (HAE) attack rates. Here we present preliminary efficacy data of patients in the United States who initiated berotralstat treatment in the real-world clinical setting and were previously on another prophylactic therapy. <h3>Methods</h3> Data was collected through the sole-source pharmacy and included patients with confirmed HAE Type I/II who actively received berotralstat 110 or 150mg from 12/16/2020-5/20/22 and were stratified by previous prophylaxis. 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 patients treated with prior prophylaxis(n=129) the median baseline attack rate was 1.7 attacks/month which decreased to 0.3(0,1.31) from days 1-90; 0.6(0,1.00) from days 91-180; 0.5(0.25,1.00) from days 181-270; and 0.5(0,1.50) from days 271-360. Specifically, in the lanadelumab group(n=53), the baseline attack rate of 1.0 attack/month decreased to 0(0,0.50) from days 1-90; 0.3(0,0.75) from 91-180; 0.5(0,1.00) from 181-270; and 0.5(0,1.38) from 271-360. In the subcutaneous C1-inhibitor group(n=31), the baseline attack rate of 1.7 attacks/month decreased to 0.8(0,2.00) from days 1-90; 0.7(0,2.08) from 91-180; 1.0(0.54,1.50) from 181-270; and 1.0(0.29,2.17) from 271-360. Patients without prior prophylaxis(n=112) had similar attack rate reductions. The most common adverse events reported were consistent with clinical trials. <h3>Conclusions</h3> Patients previously treated with another prophylactic therapy reported consistently low HAE attack rates when treated with once daily berotralstat.