BioCryst Pharmaceuticals Inc. said Japan’s Ministry of Health, Labor and Welfare approved its oral prophylactic therapy ORLADEYO (berotralstat) for pediatric patients aged 2 to under 12 with hereditary angioedema (HAE) due to C1-inhibitor deficiency.
The approval covers once-daily oral granules that can be swallowed with water or milk or sprinkled over soft, non-acidic food. BioCryst’s partner OrphanPacific Inc. holds the marketing authorization and will lead the commercial launch pending completion of Japan’s National Health Insurance System reimbursement pricing process.
The decision follows interim results from the APeX-P clinical trial, which assessed pharmacokinetics, safety and efficacy in 29 patients aged 2 to under 12. The drug was generally well-tolerated with a consistent safety profile, and nasopharyngitis was the most commonly reported treatment-emergent adverse event.
Japan’s approval expands ORLADEYO’s use in the country, where the capsule formulation has been available since January 2021 for adults and pediatric patients 12 years and older. The U.S. FDA cleared the capsule in December 2020 and the oral pellet formulation for the same pediatric group in December 2025. The drug is now approved in more than 45 countries.
BioCryst has also filed applications with the European Medicines Agency and Health Canada for use in patients aged 2 to under 12 years.
