BioCryst Announces Positive Results from APeX-P Trial for ORLADEYO® (berotralstat) in Pediatric Patients with Hereditary Angioedema Aged 2 to

Core Insights - BioCryst Pharmaceuticals announced positive interim results from the APeX-P clinical trial for ORLADEYO, showing significant reductions in HAE attack rates in pediatric patients aged 2 to <12 years [1][2][7] - The trial is the largest to date evaluating a prophylactic therapy for HAE in this age group, with data to be presented at the 2025 AAAAI/WAO Joint Congress [2][17] - ORLADEYO demonstrated safety and tolerability, with no new safety signals identified, and resulted in early and sustained reductions in monthly attack rates [7][18] Clinical Trial Results - APeX-P included a 12-week standard-of-care treatment period followed by an open-label ORLADEYO treatment period lasting up to 144 weeks, with interim data from 17 participants who completed at least 48 weeks of treatment [7][14] - The median monthly attack rate decreased from 0.96 attacks/month during the standard-of-care period to 0 attacks/month after one month of ORLADEYO treatment, maintaining a median of 0 through month 12 [7][14] - Participants with ≥5 baseline attacks/month experienced the largest reductions, with 6.20 fewer attacks/month at 12 months [14] Real-World Evidence - New real-world evidence presented at the congress showed statistically significant HAE attack rate reductions in patients with C1-INH deficiency and normal C1-INH levels after starting ORLADEYO [5][14] - Patient-reported outcomes indicated high satisfaction with ORLADEYO, with many patients willing to switch from other long-term prophylaxis therapies [12][23] - The study highlighted that 83% of participants experienced symptom onset before six years of age, and 90% were diagnosed within the same timeframe [7] Patient Insights - Patients reported a willingness to switch long-term prophylaxis therapies, with factors such as treatment burden and administration preference influencing their decisions [12][23] - Most participants reported less frequent and less severe attacks after starting ORLADEYO, with high satisfaction rates regarding their treatment [12][23] - The study included insights from U.S. patients who had been treated with ORLADEYO, showing a mean age of 43.2 years and a mean of 13.4 years since diagnosis [23]