BioCryst gets Japan approval for HAE drug in young children

BioCryst Pharmaceuticals Inc. has announced that Japan's Ministry of Health, Labor and Welfare has approved ORLADEYO (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema aged 2 to under 12 years. The drug, which is administered as a granule formulation, was approved based on interim results from the APeX-P clinical trial.

ORLADEYO demonstrated a consistent safety profile across this age group, with the most commonly reported adverse event being nasopharyngitis. The approval follows the FDA's approval of the capsule formulation for adult and pediatric patients 12 years and older in December 2020, and the oral pellet formulation's approval for U.S. pediatric patients in December 2025.

BioCryst will launch ORLADEYO for children in Japan following completion of the Japanese National Health Insurance System reimbursement pricing process.

Brief written by urgent.news from Investing.com's own syndicated text. Machine-written — may contain errors; check the original before relying on it.