BioCryst Unveils Data Showing ORLADEYO Cuts HAE Attacks in Children in Yearlong Trial

Reuters
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BioCryst Unveils Data Showing ORLADEYO Cuts HAE Attacks in Children in Yearlong Trial

BioCryst Pharmaceuticals Inc. has announced new data from the ongoing APeX-P clinical trial evaluating ORLADEYO® (berotralstat) in pediatric patients with hereditary angioedema $(HAE)$ aged 2 to under 12 years. The results, presented at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) in November 2025, show early and sustained reductions in monthly HAE attack rates over one year of treatment with once-daily ORLADEYO oral granules. The company also highlighted findings on the negative psychosocial impact of HAE and emergency care visits on children and their caregivers. A New Drug Application for ORLADEYO oral granules as prophylaxis in this pediatric population is currently under review by the U.S. Food and Drug Administration, with a decision expected by the end of 2025. If approved, ORLADEYO would be the first targeted oral prophylactic therapy for HAE patients aged 2 to under 12 years.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. BioCryst Pharmaceuticals Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9570032-en) on November 06, 2025, and is solely responsible for the information contained therein.

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