BioCryst (BCRX) Pharmaceuticals announced new data demonstrating the early and negative psychosocial impact of hereditary angioedema, HAE, and resulting emergency department, ED, and hospital visits on pediatric patients and their caregivers, as well as new one-year data from the ongoing APeX-P clinical trial showing early and sustained reductions in monthly attack rates over one year in pediatric patients with HAE aged 2 to less than12 years treated with once-daily ORLADEYO. Findings: ORLADEYO demonstrated early and sustained attack rate reduction, with 65.5% of patients attack-free at month 1 and 70.4% of patients attack-free at month 12. Median attack rate was 0 beginning at month 1 and remained 0 for 11 out of 12 months. ORLADEYO demonstrated high continuation rates: nearly all patients completed greater than or equal to48 weeks of treatment with ORLADEYO. HAE attacks start early in life: median age at diagnosis was 2.0 years with most experiencing HAE symptoms before age 6. ORLADEYO was safe and well tolerated, with no new safety signals identified. The most commonly reported treatment-emergent adverse event was nasopharyngitis. Please see below for full safety information for ORLADEYO
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