Japan Approved Orladeyo for Under-Twelves in an Open-Label Interim Analysis of 29 Children
- Japan’s MHLW granted marketing approval on 25 August 2026 for once-daily ORLADEYO (berotralstat) as prophylaxis in paediatric hereditary angioedema patients aged 2 to under 12. It arrives as a granule formulation, sprinkle-like in size, poured into the mouth with water or milk or over soft non-acidic food.
- The approval rests on interim results from APeX-P, an ongoing open-label study in patients with HAE due to C1-inhibitor deficiency. Participants (n=29) were placed in four cohorts by baseline body weight, with attack rates compared between 12 weeks of standard of care and 48 weeks of ORLADEYO. The most common treatment-emergent adverse event was nasopharyngitis.
- This is the second Japanese approval for the molecule. The capsule was approved by MHLW in January 2021 for patients 12 and older, and the same granule formulation cleared the FDA in December 2025. ORLADEYO is now approved in more than 45 countries, with filings for this age band lodged with the EMA and Health Canada.
- The product is not yet available. BioCryst will launch for children in Japan only after the National Health Insurance reimbursement pricing process completes. OrphanPacific is BioCryst’s representative partner in Japan and holds the marketing authorisation.
CI read
The molecule is not new in Japan. It has been approved there since 2021. What is new is a formulation a two-year-old can swallow, and that formulation is the entire commercial move.
- Formulation is the lifecycle lever here, and the playbook is already running in parallel. Capsule cleared Japan in 2021 for ages 12 and up. The granule opens ages 2 to under 12. FDA approved the same formulation in December 2025, with EMA and Health Canada filings lodged. One molecule, one new age band, four jurisdictions.
- The evidence is 29 children in an open-label study split four ways. The comparison is within-patient, 12 weeks of standard care against 48 weeks of drug, and it is an interim analysis of a trial still running. BioCryst’s own risk factors note that interim results do not necessarily predict final ones.
- Approval is not availability, and BioCryst does not hold the authorisation. Launch waits on NHI pricing, and OrphanPacific holds the Japanese marketing authorisation. Against ORLADEYO net revenue of $158.2 million in the second quarter alone, this band is incremental. Track the NHI listing date, not the approval.