BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX), a US-based biotechnology company focused on treatments for hereditary angioedema (HAE) and other rare diseases, said on Friday that it has received US Food and Drug Administration approval for an oral pellet formulation of once-daily ORLADEYO (berotralstat) for prophylactic use in patients aged 2 to under 12 years.

The therapy becomes the first targeted oral prophylactic option for this age group, offering an alternative to existing intravenous and subcutaneous treatments that can be burdensome for young patients. The pellet formulation is designed for child-friendly administration, either taken directly with water or milk or sprinkled over soft, non-acidic food.

The approval is supported by interim data from the APeX-P trial, the largest long-term prophylactic study in this paediatric population, which showed early and sustained reductions in monthly attack rates and a safety profile consistent with earlier trials. Nasopharyngitis was the most commonly reported treatment-emergent adverse event.

ORLADEYO is already approved in capsule form for patients aged 12 and older in the United States and more than 45 other countries. BioCryst has also submitted applications for the pellet formulation to the European Medicines Agency and the Japan Pharmaceutical and Medical Devices Agency, with additional global filings planned.