Advances in pharmacotherapy for children aged 2 to <12 years with hereditary angioedema: what is the current state of play?

INTRODUCTION: Hereditary angioedema (HAE) is a rare, potentially life-threatening disorder characterized by recurrent episodes of bradykinin-mediated angioedema. While HAE symptoms generally emerge in childhood, treatment guidelines for pediatric patients are constrained by a lack of clinical trial data - a consequence of disease rarity and of the significant ethical and logistical barriers inherent to pediatric research. Consequently, many treatment recommendations have historically relied on extrapolation from adult and adolescent studies. AREAS COVERED: We reviewed published literature and registered clinical trials evaluating pharmacological therapies (acute treatment, short-term prophylaxis, and long-term prophylaxis) in children aged 2 to <12 years with HAE, alongside ongoing pediatric clinical investigation programs. PubMed, Ovid MEDLINE databases and ClinicalTrials.gov were searched, with the final search performed on 30 July 2026. EXPERT OPINION: Dedicated pediatric clinical trials have substantially expanded the evidence base for HAE management in recent years. Established C1-inhibitor concentrates are now complemented by novel targeted therapies (icatibant, lanadelumab, berotralstat, sebetralstat, garadacimab, and donidalorsen) showing favorable efficacy or progressing through pediatric evaluation. However, the strength of evidence remains limited by small sample sizes, open-label methodologies, and disease rarity. Further pediatric-specific trials and long-term follow-up studies are needed to optimize evidence-based management in this vulnerable patient population.