BioCryst presents real-world data on ORLADEYO

BioCryst Pharmaceuticals announced new analyses of real-world use of oral, once-daily ORLADEYO that showed patients who initiated ORLADEYO experienced rapid, substantial and sustained reductions in attack rates through 18 months of treatment regardless of the severity of their disease, their history of prior prophylaxis or their C1-inhibitor level and function. Presentations: Berotralstat Prophylaxis Reduces HAE Attack Rates Regardless of Baseline Attacks: Real-World Outcomes; Patients with C1-INH deficiency who received long-term prophylaxis with ORLADEYO achieved rapid reduction in patient-reported monthly HAE attack rates from baseline, regardless of the severity of their disease. Median attack rates decreased below baseline in the first 90 days of ORLADEYO treatment and remained below baseline across all additional 90-day intervals for up to 18 months. Additionally, when stratified by baseline attacks, median monthly attack rates decreased and remained below baseline for up to 540 days on ORLADEYO regardless of their attack rate at baseline. Consistently Low Hereditary Angioedema Attack Rates with Berotralstat Regardless of Prior Prophylaxis: Real-World Outcomes; Patients who initiated ORLADEYO and were previously on at least one other prophylactic therapy at some point during their lifetime also experienced rapid, substantial and sustained reductions in HAE attack rates regardless of prior prophylactic therapy, including lanadelumab, subcutaneous C1-INH and androgens. Of note, the median monthly attack rate for patients who were previously treated with lanadelumab decreased from 1.00 at baseline to 0.33 at Days 1-90 after initiating ORLADEYO and remained below baseline through Days 451-540. Real-World Effectiveness of Berotralstat in HAE With and Without C1-Inhibitor Deficiency; Similarly, a reduction in monthly HAE attack rates was observed in patients with HAE who have normal C1-INH level and function and those with C1-INH deficiency upon initiating ORLADEYO. Patients with normal C1-INH had a median attack rate at baseline of 3.00 attacks/month, which was reduced to 1.00 at Days 1-90 and remained below baseline through Days 451-540, while patients with C1-INH deficiency had a median attack rate at baseline of 1.33 attacks/month, which was reduced to and remained at 0.50 through Days 451-540.