TOKYO, Japan — August 25, 2026
BioCryst Pharmaceuticals announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted marketing approval for ORLADEYO® (berotralstat) as a once-daily prophylactic treatment for pediatric patients aged 2 to under 12 years with hereditary angioedema (HAE). The regulatory decision expands access to an oral preventive treatment for children with HAE and represents the second ORLADEYO approval in Japan, following the earlier approval of the capsule formulation for patients aged 12 years and older. BioCryst plans to launch the pediatric formulation after completion of Japan’s National Health Insurance reimbursement pricing process.
Pediatric HAE Treatment Gains an Oral Option in Japan
The Japanese approval is significant because children with HAE have traditionally faced treatment approaches involving injections, creating an additional administration burden for patients and caregivers. ORLADEYO’s pediatric granule formulation, known as the pellet formulation in the United States, was developed around the administration needs of younger children. The formulation can be taken directly with water or milk or sprinkled over a spoonful of soft, non-acidic food. By extending once-daily oral prophylaxis to children between 2 and under 12 years of age, the approval could provide families with a more convenient approach to preventing recurrent HAE attacks. ORLADEYO works by reducing plasma kallikrein activity, targeting a key component of the pathway involved in HAE attacks
APeX-P Data Support Pediatric Regulatory Decision
The approval was supported by interim findings from APeX-P, an ongoing open-label clinical study evaluating the pharmacokinetics, safety and efficacy of ORLADEYO in children aged 2 to under 12 years with HAE caused by C1-inhibitor deficiency. The study enrolled 29 pediatric patients across four body-weight cohorts and compared HAE attack rates requiring on-demand treatment during a 12-week standard-of-care period with outcomes during 48 weeks of ORLADEYO treatment. Interim results showed early and sustained reductions in monthly HAE attack rates, while ORLADEYO was generally well tolerated and maintained a safety profile consistent with previous clinical experience in older patients. No new safety signals were identified in the interim analysis, with nasopharyngitis reported as the most common treatment-emergent adverse event. The pediatric data add clinical support for expanding preventive treatment options in a population where reducing attack frequency and treatment burden remains an important priority.
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BioCryst Expands Global Pediatric HAE Access
The Japanese decision strengthens BioCryst’s international expansion of ORLADEYO and adds another regulatory milestone for the pediatric formulation. ORLADEYO is now approved in more than 45 countries, while the U.S. approved its pediatric oral pellet formulation in December 2025 for children aged 2 to under 12 years. BioCryst has also submitted applications for pediatric use to the European Medicines Agency and Health Canada, with additional regulatory filings planned in other territories. In Japan, the company will work with OrphanPacific, Inc., its representative partner and marketing authorization holder, to prepare the pediatric launch following the national reimbursement pricing process. The latest approval reinforces BioCryst’s focus on rare-disease medicines and oral prophylactic treatment for HAE, while extending ORLADEYO’s reach into younger patient populations.
Source:BioCryst Pharmaceuticals,press relese
