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BioCryst wins Japan approval for pediatric ORLADEYO
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BioCryst wins Japan approval for pediatric ORLADEYO

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  • BioCryst (NASDAQ:BCRX) received approval in Japan for pediatric use of once-daily ORLADEYO (berotralstat) in hereditary angioedema patients.
  • The approval covers a granule formulation for children aged 2 to under 12 years.
  • The decision follows interim APeX-P trial data showing reduced monthly attack rates and a safety profile consistent with older patients.

BioCryst (NASDAQ:BCRX) announced that Japan’s Ministry of Health, Labor and Welfare approved once-daily ORLADEYO (berotralstat) as a preventive treatment for pediatric hereditary angioedema (HAE) patients aged 2 to under 12 years.

The approval includes a sprinkle-like granule formulation designed for children and follows interim data from the APeX-P clinical trial, which showed reductions in monthly HAE attack rates.

The APeX-P trial showed early and sustained reductions in monthly attack rates, with a safety profile consistent with previous ORLADEYO experience in older patients.

OrphanPacific holds marketing authorization for ORLADEYO in Japan, and the approval represents the second ORLADEYO approval in the country following previous capsule authorization.

BioCryst stated that ORLADEYO has also received approvals in the United States and more than 45 other countries for hereditary angioedema prevention.

The company develops oral and small-molecule medicines for rare diseases, including hereditary angioedema, with ORLADEYO as one of its commercial products.

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