BioCryst to Present New ORLADEYO® (berotralstat) Results from APeX-P Pediatric Trial at 2025 American Academy of Allergy, Asthma & Immunology / World Allergy Organization Joint Congress

Core Insights - BioCryst Pharmaceuticals will present five abstracts on ORLADEYO (berotralstat) at the 2025 AAAAI/WAO joint congress, focusing on its use for hereditary angioedema (HAE) [1][2] Group 1: Product Information - ORLADEYO is the first and only oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, administered as one capsule daily [3][4] - The drug functions as a plasma kallikrein inhibitor, reducing the activity of plasma kallikrein to prevent HAE attacks [3] Group 2: Clinical Trials and Research - A late-breaking abstract will present results from the APeX-P trial, which evaluates ORLADEYO in pediatric patients aged 2 to under 12 years with HAE [2] - The five abstracts will cover various aspects, including real-world attack rates before and after berotralstat initiation and patient-reported impacts on attack frequency and severity [8] Group 3: Safety and Efficacy - The safety and effectiveness of ORLADEYO for treating acute HAE attacks have not been established, and it is not recommended for such use [5][10] - Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease, occurring in 10% or more of patients [6] Group 4: Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on developing therapies for hereditary angioedema and other rare diseases, leveraging expertise in structure-guided drug design [11]