Overview
- The pediatric formulation makes Orladeyo the first and only targeted oral prophylactic option for patients with hereditary angioedema starting at age two.
- Approval is supported by interim results from the open-label APeX-P study in children that reported a consistent safety profile with early, sustained reductions in monthly attack rates and no new safety signals.
- The sprinkle-like pellets can be swallowed with water or milk or administered on a spoonful of soft, non‑acidic food to ease dosing in young children.
- Orladeyo’s capsule form has been FDA-approved since December 2020 for prophylaxis in adults and adolescents 12 years and older.
- BioCryst has filed for pediatric use with the European Medicines Agency and Japan’s PMDA and plans additional submissions, including in Canada.