BioCryst Pharmaceuticals Reports That Orladeyo Oral Granules Demonstrated Early And Sustained Reductions In HAE Attack Rates Over One Year In Pediatric Patients Aged 2 To <12 In The Ongoing APeX-P Trial

Author: Benzinga Newsdesk | November 06, 2025 08:25am

 Findings highlight negative psychosocial impact of HAE attacks and resulting ED/hospital visits on pediatric patients and their caregivers –

–  ORLADEYO oral granules demonstrated early and sustained reduction in HAE attacks over one year of treatment in second interim analysis of APeX-P –

 

–  ORLADEYO oral granules are currently under review with U.S. FDA for ages 2 to <12 with decision expected by end of year; would be first and only targeted oral prophylactic therapy for patients with HAE age 2 to <12 –

 

 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new data demonstrating the early and negative psychosocial impact of hereditary angioedema (HAE) and resulting emergency department (ED) and hospital visits on pediatric patients and their caregivers, as well as new one-year data from the ongoing APeX-P clinical trial showing early and sustained reductions in monthly attack rates over one year in pediatric patients with HAE aged 2 to <12 years treated with once-daily ORLADEYO® (berotralstat).

The New Drug Application (NDA) for an oral granule formulation of once-daily ORLADEYO as prophylaxis in pediatric patients with HAE aged 2 to <12 years is currently under review with the U.S. Food and Drug Administration (FDA), with a target action date of December 12, 2025. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. A capsule formulation of ORLADEYO was approved by the FDA in December 2020 as prophylaxis for patients with HAE aged 12 years and older.

Data are part of presentations at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is taking place in Orlando, Fla. from November 6-10, 2025.

"Our psychosocial data elucidate that HAE attacks typically begin well before puberty and can have a profoundly negative impact on the mental health and quality of life of both children and their caregivers. Currently there are only injectable treatments approved for prevention of attacks in patients under 12, and findings highlight the clear and urgent need for a safe and effective long-term prophylactic treatment with a child-friendly method of administration," said Dr. Donald S. Fong, chief medical officer of BioCryst. "ORLADEYO would be the first and only targeted oral prophylactic therapy for patients with HAE aged 2 to less than 12 years, if approved. We believe that the one-year data from APeX-P reinforce the role that ORLADEYO oral granules could potentially play in preventing attacks in patients 2 to less than 12 years, and we look forward to the FDA's decision on our application later this year."

Posted In: BCRX