BioCryst Announces Positive Results from APeX-P Trial for ORLADEYO® (berotralstat) in Pediatric Patients with Hereditary Angioedema Aged 2 to <12 Years

Core Insights - BioCryst Pharmaceuticals announced positive interim results from the APeX-P clinical trial for ORLADEYO, showing significant reductions in hereditary angioedema (HAE) attack rates in pediatric patients aged 2 to <12 years [1][2][6] - The company plans to submit a New Drug Application to the FDA within the year, addressing a significant unmet need for children with HAE [2][3] Clinical Trial Results - APeX-P is the largest trial evaluating prophylactic therapy for HAE in young patients, demonstrating that ORLADEYO is safe and well-tolerated, with early and sustained reductions in monthly attack rates [2][3] - In the standard-of-care period, the median monthly attack rate was 0.96 attacks/month, which dropped to 0 after one month of ORLADEYO treatment, maintaining a median of 0 through month 12 [6][11] - 83% of participants experienced symptom onset before six years of age, with 90% diagnosed within the same timeframe [6] Real-World Evidence - New real-world evidence presented at the 2025 AAAAI/WAO Joint Congress indicates statistically significant reductions in HAE attack rates for patients with C1-INH deficiency and normal C1-INH levels after starting ORLADEYO [4][11] - Patients with ≥5 baseline attacks/month experienced a reduction of 6.20 attacks/month at 12 months and 6.37 attacks/month at 18 months [11] - Among patients with 0 attacks/month at baseline, 70% maintained this low attack rate at 12 months [11] Patient-Reported Outcomes - Patient-reported outcomes indicate a willingness to switch long-term prophylaxis (LTP) and improved treatment satisfaction after initiating ORLADEYO [9][10] - Participants reported less frequent and severe attacks after switching to ORLADEYO, with high satisfaction rates regarding their treatment [21][22] Future Directions - BioCryst is committed to addressing the needs of HAE patients and advancing its pipeline of therapies, with ORLADEYO being the first oral therapy specifically designed for HAE prevention [15][23]