BioCryst’s New ORLADEYO Data Adds Real-World HAE Evidence
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BioCryst Pharmaceuticals is presenting new clinical data and real-world evidence for ORLADEYO (berotralstat) in hereditary angioedema. The update matters because it expands the evidence base around an FDA-approved oral prophylactic option for adults and pediatric patients 2 years and older.
BioCryst Presents New Clinical Data and real-world evidence for Orladeyo (berotralstat) at the 2026 EAACI meeting, alongside navenibart ALPHA-STAR results. The centerpiece pediatric update from APeX-P shows median on-demand–treated HAE attacks falling from 0.691 to 0.169 per month over 48 weeks in children aged 2 to under 12.
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Key Takeaways
- BioCryst disclosed Orladeyo and navenibart posters for EAACI 2026 (June 12–15, Istanbul).
- APeX-P 48-week data: median adjusted on-demand attack rate 0.691 → 0.169 attacks/month.
- Orladeyo remains an oral once-daily plasma kallikrein inhibitor for HAE prophylaxis age 2+.
- Navenibart Phase 1b/2 ALPHA-STAR subgroup data support ongoing Phase 3 evaluation.
What did BioCryst Presents New Clinical Data include?
In a June 12, 2026 GlobeNewswire release, BioCryst said it would present new Orladeyo clinical and real-world evidence plus navenibart Phase 1b/2 data at EAACI. The company positioned Orladeyo as a targeted oral prophylactic option for HAE patients aged 2 and older. Full release text is on GlobeNewswire.
What did APeX-P show at 48 weeks?
APeX-P (NCT05453968) is a Phase 3 pediatric study of once-daily berotralstat in children 2 to under 12 years. BioCryst called the updated 48-week analysis the largest long-term prophylaxis dataset in pediatric HAE for this program.
- Median adjusted attack rate needing on-demand treatment: 0.691/month in a 12-week standard-of-care window
- Median adjusted rate on Orladeyo: 0.169/month over 48 weeks
- Reported ranges spanned 0–5.03 attacks/month in the SOC period and 0–1.75 on treatment
- Trial enrollment listed as 29 pediatric participants across multiple countries on ClinicalTrials.gov
Registration details are on ClinicalTrials.gov NCT05453968.
How does labeling define Orladeyo use?
U.S. labeling describes Orladeyo as a plasma kallikrein inhibitor for prophylaxis to prevent HAE attacks in adults and pediatric patients 2 years and older. Prescribers should rely on the current label for dosing, warnings, and limitations rather than congress posters alone. Label text is available via DailyMed Orladeyo.
What else is in the HAE portfolio update?
BioCryst also highlighted navenibart, an investigational long-acting monoclonal antibody plasma kallikrein inhibitor, with Phase 1b/2 ALPHA-STAR post hoc subgroup analyses at EAACI and Phase 3 work ongoing. Real-world Orladeyo abstracts addressed adolescent and adult outcomes, including patients switching from other long-term prophylaxis. Open-label access study NCT04933721 (APeX-A) continues to provide long-term access and safety follow-up in selected countries.
What remains unproven
Congress posters and company releases are not peer-reviewed final publications. Attack-rate reductions in APeX-P are descriptive for this open-label pediatric design and should not be compared head-to-head with injectable prophylaxis trials without careful methods review. Navenibart remains investigational; Phase 3 success is not guaranteed.
Related NovaPharma coverage
- BioCryst Presents New HAE Data for ORLADEYO® and Navenibart at EAACI 2026
- BioCryst Secures $70M Upfront in European Licensing Deal for Navenibart
- European Navenibart Deal: A Strategic Win for BioCryst's Rare Disease Portfolio
Frequently Asked Questions
What did BioCryst Presents New Clinical Data cover at EAACI 2026?
On June 12, 2026, BioCryst announced new Orladeyo (berotralstat) clinical and real-world evidence plus navenibart Phase 1b/2 ALPHA-STAR data, presented across posters at the EAACI Annual Meeting in Istanbul from June 12–15, 2026.
What did APeX-P 48-week data show for pediatric Orladeyo use?
Updated APeX-P analysis in children aged 2 to under 12 years reported that the median adjusted HAE attack rate requiring on-demand treatment fell from 0.691 attacks per month in a 12-week standard-of-care period to 0.169 attacks per month over 48 weeks of Orladeyo.
What is Orladeyo indicated for?
Orladeyo (berotralstat) is an oral plasma kallikrein inhibitor indicated for prophylaxis to prevent hereditary angioedema attacks in adults and pediatric patients 2 years of age and older, per U.S. labeling.
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