- BioCryst Pharmaceuticals (BCRX, Financial) presented new data showing ORLADEYO® significantly reduces HAE attacks.
- Severe HAE patients saw a reduction from over 7 attacks per month to below 2 after ORLADEYO treatment.
- Adolescent patients experienced a reduction from over 2 attacks per month to under 1 with ORLADEYO.
BioCryst Pharmaceuticals (BCRX) recently showcased compelling real-world evidence highlighting the efficacy of their drug, ORLADEYO® (berotralstat), in the treatment of Hereditary Angioedema (HAE), presented at the 2025 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR).
The retrospective study, analyzing data from December 2020 to January 2024, focused on two key patient groups: severe HAE patients, and adolescents aged 12-17. The findings revealed significant reductions in the frequency of HAE attacks after introducing ORLADEYO into their treatment regimen.
For the severe HAE patient group consisting of 56 individuals, the study showed a drastic reduction in attack rates from a baseline of 7.78-8.23 attacks per month to just 1.24-1.90 attacks per month post-treatment. Notably, patients continued to maintain a reduction of 6.43 fewer attacks per month at the 18-month mark.
Additionally, the study observed 99 adolescent patients aged 12-17, where attack rates decreased significantly from a baseline of 2.07-2.30 attacks per month to under one attack per month, specifically between 0.36 and 0.76 attacks, representing a sustained reduction of 1.85 attacks per month at 18 months.
These results underscore ORLADEYO's effectiveness and enduring impact across varying populations, reinforcing its status as a leading oral therapy option for HAE. The continued use of ORLADEYO could improve patient quality of life by reducing the burden of frequent HAE attacks and providing a more convenient oral administration compared to injectable alternatives.
