BioCryst reports a 30% reduction in angioedema-related medical visits after switching to ORLADEYO in a real-world study of 78 patients with HAE.
Written By: Charvi Kalal, PharmD
Reviewed By: Pharmacally Editorial Team
BioCryst Pharmaceuticals reported new real-world evidence on November 13, 2026, showing reduced healthcare utilization among patients with hereditary angioedema (HAE) who received ORLADEYO (berotralstat), an oral prophylactic therapy. Two retrospective studies presented at the American College of Allergy, Asthma & Immunology (ACAAI) 2026 Annual Scientific Meeting in Phoenix, Arizona, evaluated healthcare utilization after patients either initiated long-term prophylactic therapy (LTP) or switched to ORLADEYO from another preventive treatment.
The findings provide additional evidence on healthcare utilization in routine clinical practice, with the switching analysis reporting a 30% reduction in overall angioedema-related medical visits.
Real-World Studies Evaluated Treatment Initiation and Switching
Poster R096 released ahead of its scheduled presentation on November 14 evaluated 78 patients aged 12 years and older with HAE who switched from another LTP to ORLADEYO. The retrospective pre-post study used US administrative claims data linked to BioCryst’s ORLADEYO patient base through its sole-source specialty pharmacy. Patients were required to have at least six months of continuous insurance eligibility before starting ORLADEYO.
A separate analysis, presented on November 13 as Poster R077, examined healthcare utilization among patients initiating LTP for the first time using US claims data spanning June 2017 through October 2024. This retrospective observational study included 128 patients starting ORLADEYO, 236 starting lanadelumab, and 95 starting subcutaneous plasma-derived C1-inhibitor (SC-pdC1-INH). Patients were aged 12 years and older, had documented HAE before treatment initiation, and maintained at least six months of continuous insurance enrollment before and after the index date.
Both studies assessed changes in all-cause and angioedema-related hospitalizations and emergency department (ED) visits before and after prophylactic treatment initiation.
Switching to ORLADEYO Reduced Angioedema-Related Medical Visits
In the switching cohort (Poster R096), overall angioedema-related medical visits decreased by 30% per patient-year (PPY) after patients transitioned to ORLADEYO (). Angioedema-related outpatient and ED visits also declined by 30% PPY ().
Angioedema-related hospitalizations decreased to 0.12 PPY after treatment initiation, corresponding to a rate ratio of 0.54 (95% confidence interval [CI]: 0.21–1.39). However, this reduction was not statistically significant, as the confidence interval crossed 1. The distinction is notable because the reported 30% reduction in overall and outpatient/ED visits reached
statistical significance, whereas the hospitalization findings did not establish a significant reduction.
Healthcare Utilization Declined Across Three Prophylactic Therapies
In the treatment-initiation study (Poster R077), all three cohorts experienced statistically significant reductions in ED visits, alongside numerical reductions in hospitalizations:
- All-cause hospitalizations: Declined by 26% to 53% across cohorts (53% in the ORLADEYO group).
- All-cause ED visits: Decreased by 34% to 48%.
- Angioedema-related hospitalizations: Fell by 46% to 59%.
- Angioedema-related ED visits: Declined by 43% to 61% (61% in the ORLADEYO group).
No statistically significant differences in the magnitude of reductions were reported between patients initiating ORLADEYO, lanadelumab, or SC-pdC1-INH.
ORLADEYO: Mechanism and Safety Considerations
ORLADEYO is an oral plasma kallikrein inhibitor indicated for prophylaxis to prevent HAE attacks in adults and pediatric patients aged 2 years and older. By inhibiting plasma kallikrein activity, berotralstat downregulates the pathway driving bradykinin-mediated angioedema attacks. It is not indicated for treating acute HAE attacks.
The recommended dose for adults and patients aged 12 years and older is 150 mg once daily with food, while children aged 2 to under 12 years receive weight-based oral pellet doses. Prescribing information includes precautions regarding QTc prolongation at higher-than-recommended doses, gastrointestinal adverse reactions, and drug interactions involving P-glycoprotein, CYP2D6, and CYP3A4. These safety parameters are derived from approved labeling and were not evaluated as endpoints in the two real-world studies.
Clinical Interpretation: Healthcare Utilization Evidence Requires Cautious Reading
The findings suggest that healthcare utilization declined after patients initiated long-term prophylaxis, while patients switching to ORLADEYO experienced fewer angioedema-related medical visits. The 30% reduction in the switching cohort is clinically relevant because it captures real-world healthcare use beyond clinical trial measures of attack frequency.
Nevertheless, both studies used retrospective pre-post designs without randomization or concurrent control groups. Observed reductions may partly reflect regression to the mean, particularly when patients initiate or change prophylaxis following periods of frequent attacks. Furthermore, the lack of statistically significant differences between the three therapies in the initiation study should not be interpreted as evidence of therapeutic equivalence or superiority.
Several additional methodological limitations warrant consideration
- Cohort size and prior therapy: The switching analysis involved a relatively small sample of 78 patients, and the company announcement did not specify reasons for switching or details of prior prophylactic regimens.
- Claims data constraints: Administrative claims cannot capture HAE attack frequency, symptom severity, quality of life, or treatment safety, and therefore cannot establish that reduced healthcare use directly reflects improved disease control.
- Data source and publication status: The switching analysis relied on data linked to BioCryst’s sole-source specialty pharmacy, and both studies remain conference poster presentations rather than peer-reviewed publications. Additionally, no cost-effectiveness or healthcare expenditure data were provided to substantiate potential economic benefits.
Overall, these data add real-world support for ORLADEYO as a long-term prophylactic option, particularly for patients transitioning from another preventive therapy. Further peer-reviewed and prospective evidence is needed to determine how closely reductions in healthcare utilization correlate with sustained improvements in disease control and patient outcomes.
Reference
BioCryst Presents New Real-World Evidence Showing Reduced Angioedema-Related Medical Visits After Switching to ORLADEYO® (berotralstat) at ACAAI 2026 Annual Scientific Meeting, BioCryst Pharmaceuticals, 13 November 2026
About the Writer
Charvi Kalal (LinkedIn) is a Pharm.D intern and aspiring medical writer with hands-on experience in medical writing, clinical research, pharmacovigilance, and clinical pharmacy. She has experience in medical content writing through CliMed, along with exposure to ADR monitoring, prescription analysis, patient counselling, literature review, and research documentation. Her research focuses on ADR monitoring and reporting in breast cancer patients receiving targeted therapy. With certifications in GCP, biomedical research, scientific writing, and clinical research, she is passionate about transforming scientific evidence into accurate, engaging, and accessible healthcare content.