BioCryst Pharmaceuticals announced real-world evidence demonstrating significant and sustained reductions in HAE attack rates for adolescents and individuals with severe HAE taking ORLADEYO (berotralstat) for up to 18 months. The data, presented at the 2025 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR), comes from a retrospective pre-post study utilizing data collected from BioCryst’s sole-source pharmacy between December 2020 and January 2024. The study focused on the impact of ORLADEYO on two specific HAE patient populations: those with severe disease (experiencing eight or more attacks per month) and adolescents aged 12-17.
This real-world evidence is crucial for solidifying ORLADEYO’s position in the HAE treatment landscape. It provides clinicians and patients with further confidence in the drug’s long-term effectiveness, especially for those with severe disease or adolescent patients who often face unique challenges in managing their condition. Demonstrating sustained efficacy over 18 months is particularly important, as it suggests ORLADEYO can offer consistent, long-term control of HAE attacks, potentially improving patients’ quality of life and reducing the burden of this chronic condition.
The study showed significant reductions in HAE attack rates in both patient groups. Adults with severe HAE experienced a decrease of 6.25 attacks per month at 12 months and 6.43 attacks per month at 18 months compared to baseline. Adolescents saw reductions of 1.56 attacks per month at 12 months and 1.85 attacks per month at 18 months compared to baseline. These consistent reductions across different follow-up periods emphasize the drug’s sustained effectiveness.
This positive real-world data strengthens ORLADEYO’s market potential and further validates its use as a prophylactic treatment for HAE. The demonstrated efficacy in both adolescents and those with severe HAE could lead to increased adoption by physicians, ultimately benefiting a broader range of patients seeking effective, long-term HAE attack prevention. The continued generation of real-world evidence will be key to solidifying ORLADEYO’s place as a standard of care in HAE management.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
