BioCryst presents new real-world evidence on ORLADEYO in severe HAE

BioCryst (BCRX) Pharmaceuticals announced new real-world evidence on the use of oral, once-daily ORLADEYO in adolescents and people with severe HAE showing significant and sustained reductions in HAE attack rates through 18 months of follow-up after beginning treatment with ORLADEYO in both patient populations. The real-world evidence was presented in two posters at the 2025 International Society for Pharmacoeconomics and Outcomes Research conference, which is being held in Montreal from May 13-16, 2025. Significant and sustained reductions in attack rates after ORLADEYO initiation: The results presented in two posters at ISPOR 2025 were from a retrospective pre-post study using outcomes collected from BioCryst’s sole-source pharmacy from December 15, 2020, to January 8, 2024. The poster “Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Patients with C1 Inhibitor Deficiency and greater than or equal to8 Attacks/month” detailed findings from 56 U.S. patients with HAE with C1-inhibitor deficiency who received ORLADEYO. Patients experienced significantly lower HAE attack rates while on ORLADEYO in each 90-day follow-up period compared to baseline. Patients experienced significantly fewer HAE attacks per month following ORLADEYO initiation during every 90-day follow-up period relative to baseline, including: 6.25 fewer attacks/month at 12 months; 6.43 fewer attacks/month at 18 months. The poster “Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Adolescents” highlighted outcomes reported from 99 U.S. patients with HAE aged 12-17 years who received ORLADEYO. Patients had significantly lower HAE attack rates while on ORLADEYO during each 90-day follow-up period compared to the mean baseline rate. Compared to baseline, adolescents experienced statistically significant and sustained reductions in HAE attack rates after ORLADEYO initiation during each 90-day follow-up period, including: 1.56 fewer attacks/month at 12 months; 1.85 fewer attacks/month at 18 months.