Data from a real-world clinical trial shows effectiveness of berotralstat in reducing rates of HAE attacks in adolescents and people with severe HAE
The results of two clinical trials, in which berotralstat (Orladeyo) was tested in adolescents and people with severe HAE, were provided by the pharmaceutical company BioCryst. The key updates shared with HAEi were:
Patients with severe HAE
- Patients experienced significantly lower HAE attack rates while on berotralstat in each 90-day follow-up period (1.24-1.90 attacks/month) compared to baseline (7.78-8.23 attacks/month).
- Patients experienced significantly fewer HAE attacks per month following berotralstat initiation during every 90-day follow-up period relative to baseline.
Adolescents
- Adolescent patients had significantly lower HAE attack rates while on berotralstat during each 90-day follow-up period (0.36-0.76 attacks/month) compared to the mean baseline rate (2.07-2.30 attacks/month).
- Compared to baseline, adolescents experienced statistically significant and sustained reductions in HAE attack rates after berotralstat initiation during each 90-day follow-up period.
Dr. Raffi Tachdjian, Associate Clinical Professor of Medicine & Pediatrics, Division of Allergy & Clinical Immunology, David Geffen School of Medicine, said: “The outcomes detailed in these posters show how Orladeyo is making a difference for people living with HAE, in particular those with very severe disease and those who are adolescents. These two groups experienced far fewer attacks per month compared to baseline after starting Orladeyo. These kinds of real-world results should give physicians as well as their HAE patients the additional confidence to improve control of their attacks.”
These data were presented in two posters at the 2025 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR), which was held in Montreal from 13-16 May 2025.
(Source: BioCryst)