In a recent press release, BioCryst Pharmaceuticals has shared that it received approval from Japan’s Ministry of Health, Labour and Welfare for the use of ORLADEYO® (berotralstat) as a preventive treatment in children aged 2 to younger than 12 years with hereditary angioedema (HAE). The decision expands access to the oral therapy for younger patients in Japan, providing an alternative to injectable prophylactic treatments that have traditionally been used in this population.
HAE is a rare genetic disorder characterized by recurrent episodes of swelling that can affect various parts of the body, including the skin, gastrointestinal tract, and airways. Managing the condition often requires long-term preventive treatment to reduce the frequency and severity of attacks.
The newly approved pediatric formulation was designed with children in mind. The granules can be taken orally either directly with water or milk or mixed with a small amount of soft, non-acidic food, potentially simplifying administration for patients and caregivers compared with injection-based therapies.
The approval was supported by interim findings from the Phase 3 APeX-P study, which BioCryst describes as the largest clinical trial evaluating long-term prophylaxis in pediatric patients with HAE. The study assessed pharmacokinetics, safety, and efficacy in children aged 2 to younger than 12 years with HAE caused by C1-inhibitor deficiency.
According to data presented at scientific meetings and published in Annals of Allergy, Asthma & Immunology, treatment with berotralstat was generally well tolerated and produced sustained reductions in monthly HAE attack rates. Investigators reported a safety profile consistent across the pediatric age range, with no new safety concerns identified beyond those already observed in studies involving adolescents and adults. Nasopharyngitis was the most frequently reported treatment-emergent adverse event.
BioCryst said it expects to make the treatment available in Japan after reimbursement pricing is finalized through the country’s National Health Insurance system. OrphanPacific, Inc., BioCryst’s commercial partner in Japan, holds the local marketing authorization.
The latest authorization represents the second approval for ORLADEYO in Japan. The capsule formulation was cleared in 2021 for preventive treatment in adults and children aged 12 years and older. Globally, ORLADEYO is approved in more than 45 countries, and the oral pellet formulation received U.S. approval in December 2025 for pediatric patients aged 2 to younger than 12 years.
BioCryst is also seeking additional regulatory approvals for the pediatric indication in other regions. Applications are currently under review by the European Medicines Agency and Health Canada, with further submissions planned in additional markets. The company says these efforts are part of a broader strategy to expand access to oral preventive treatment options for younger patients living with HAE worldwide.
