A once-daily oral treatment is showing real promise for kids with hereditary angioedema. Updated data from the APeX-P study found that berotralstat (Orladeyo) significantly reduced HAE attacks requiring on-demand treatment in children aged 2–12, with benefits appearing as early as week 4. It's the first and only oral prophylactic option approved for this age group.
A once-daily oral treatment is making a meaningful difference for young children living with hereditary angioedema (HAE). Updated 48-week interim data from the ongoing APeX-P study, presented at the 2026 European Academy of Allergy and Clinical Immunology Annual Congress, show that berotralstat (Orladeyo, BioCryst) significantly reduced HAE attacks requiring on-demand treatment in children aged 2 to under 12 years — with reductions appearing as early as week 4.
The study followed 29 pediatric patients across four weight-based dosing cohorts, receiving either a capsule or oral pellet formulation. Notably, the FDA approved berotralstat oral pellets for this age group in December 2025, making it the first and only oral prophylactic HAE treatment for young children — a major step given that injections have historically been the standard.
By the Numbers:
Why it matters: HAE can strike early in life — this study's patients had a mean symptom onset age of just 2.9 years. A safe, effective, child-friendly oral option could dramatically reduce treatment burden for young patients and their caregivers.
