FDA Clears BioCryst's ORLADEYO Pellet Formulation for Young HAE Patients, Marking Major Pediatric Milestone

BioCryst Pharmaceuticals has received U.S. Food and Drug Administration clearance for a pediatric formulation of ORLADEYO (berotralstat), establishing the company’s medication as the leading targeted oral preventive option for hereditary angioedema in children aged 2 and beyond.

The latest regulatory approval marks a significant expansion of ORLADEYO’s clinical footprint. The newly approved oral pellet format has been specifically designed with pediatric patients in mind, offering a more practical administration method compared to traditional treatment approaches for this young patient population.

The clinical foundation for this approval comes from the APeX-P trial, which demonstrated meaningful clinical benefits for the targeted age group. Study data revealed that patients experienced prompt and sustained decreases in their monthly HAE attack frequency, providing evidence of the treatment’s preventive effectiveness in reducing disease burden for children.

With this clearance, ORLADEYO now holds a unique market position as the first and only targeted oral prophylactic medication available across all HAE patient ages starting from 2 years old. This approval underscores BioCryst’s commitment to addressing treatment gaps in pediatric rare disease management and provides families with an evidence-based preventive therapy option tailored specifically for younger patients experiencing hereditary angioedema.