BioCryst Pharmaceuticals (NASDAQ: BCRX) announced that Japan's Ministry of Health, Labor and Welfare has granted marketing approval for ORLADEYO® (berotralstat) as a once-daily prophylactic therapy for pediatric patients with hereditary angioedema (HAE) aged 2 to under 12 years.

The approval covers the granule formulation of ORLADEYO, which can be swallowed directly with water or milk, or sprinkled over soft, non-acidic food. The decision is based on interim results from the APeX-P clinical trial, which evaluated pharmacokinetics, safety, and efficacy in 29 patients aged 2 to under 12 years with HAE due to C1-inhibitor deficiency. Interim results showed the drug was generally well tolerated with a consistent safety profile, and produced early and sustained reductions in monthly attack rates. The most commonly reported treatment-emergent adverse event was nasopharyngitis.

BioCryst said it will launch ORLADEYO for this age group in Japan following completion of the National Health Insurance reimbursement pricing process. OrphanPacific, Inc. holds the marketing authorization in Japan as BioCryst's representative partner.

This marks the second regulatory approval for ORLADEYO in Japan. The capsule formulation was approved by the MHLW in January 2021 for adults and pediatric patients 12 years and older. In December 2025, the U.S. Food and Drug Administration approved the pellet formulation for the same pediatric age group in the United States.

BioCryst has filed applications for this age group with the European Medicines Agency and Health Canada, with additional regulatory filings planned in other markets.