BioCryst Pharmaceuticals, Inc. announced new real-world data demonstrating rapid, sustained reduction of patient-reported HAE attacks and consistently low attack rates among patients 12 years and older who started on oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE), including patients who switched from other prophylactic therapies. The presentations at ACAAI are based on analyses from patient-reported results collected in the real-world clinical setting from BioCryst's sole-source pharmacy, including HAE Type I and Type II patients in the United States who actively received ORLADEYO between December 16, 2020, and May 20, 2022. Consistently Low Hereditary Angioedema Attack Rates Observed with Berotralstat Regardless of Previous Prophylaxis: Real-World Outcomes (poster #P062); Saturday, November 12, 11:35 a.m. ET; Monitor 10, Exhibit Hall This analysis assessed patient-reported HAE attack rates of patients on ORLADEYO 110 mg or 150 mg who were previously on another prophylactic therapy (n=129), including lanadelumab (n=53), a subcutaneous (SC) C1 esterase inhibitor (n=31), danazol (n=15) and an intravenous (IV) C1 esterase inhibitor (n=21).

Nine patients were on a combination of prophylactic therapies. Regardless of prior prophylaxis, a rapid reduction in median attack rates was observed early (1.67 attacks/month at baseline to a median attack rate of 0.33 attacks month in days 1-90). The reduction of median attack rates was sustained throughout the 360-day treatment period.

Upon initiating ORLADEYO treatment, the reduction in median attack rates from baseline over the 1–360 days period was consistent for patients regardless of their prior prophylaxis therapy. The reductions after starting ORLADEYO for each prior prophylactic therapy were: 77% reduction for patients previously on lanadelumab; 64 percent reduction for patients previously on SC C1-INH; 70% reduction for patients previously on danazol; and 72% reduction previously on IV C1-INH. The incidence of AEs reported was lower than the incidence reported in clinical trials.