BioCryst Highlights Real-world Data Showing ORLADEYO® (berotralstat) Consistently Reduces HAE Attacks Across All Ages

Core Insights - New data presented at the 14th C1-inhibitor Deficiency & Angioedema Workshop supports the use of berotralstat (ORLADEYO) for hereditary angioedema (HAE) across all age groups, demonstrating significant efficacy and safety [1][2] Group 1: Efficacy and Safety of Berotralstat - The APeX-P trial showed a reduction in the percentage of days with angioedema symptoms among young children, with a drop from a mean of 11% to 4% over 12 weeks, sustained up to 48 weeks [4] - In the Italian Expanded Access Program, median Angioedema Control Test scores improved from 11.5 to 16.0 over six months, indicating better disease control [6] - The Berolife study reported a decrease in the median monthly attack rate from 2.25 at baseline to 0.55 after six months of treatment in adolescents [10][11] Group 2: Patient Demographics and Treatment Cohorts - The APeX-P study involved children aged 2 to 11 years, with a median age of 8 years and a mean of 18 missed school days annually due to HAE prior to treatment [4] - The Italian Expanded Access Program included 22 patients receiving berotralstat free of charge, with 15 transitioning to commercially available ORLADEYO upon program closure [5][7] Group 3: Treatment Administration and Mechanism - ORLADEYO is the first oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, administered as one capsule daily [12] - The treatment works by decreasing the activity of plasma kallikrein, which is involved in the pathophysiology of HAE [12]