Two phase III trials of new drugs for eosinophilic oesophagitis (EoE) have been reported in The New England Journal of Medicine. The findings not only highlight new treatments for the condition, but also potentially shift the emphasis for future therapeutic targets and diagnostic criteria.

In the first trial (EoE KIDS), Mirna Chehade and colleagues assessed dupilumab, a human monoclonal antibody that blocks IL-4 and IL-13 pathways, for EoE in patients aged 1–11 years. “Dupilumab was approved in the US to treat children with EoE 12 years and older in May 2022. FDA approval was then extended to treat children 1 year and older weighing at least 15 kg in January 2024,” explains Chehade, who adds that these new results provide the basis of this recent FDA approval. In this phase III trial, paediatric patients with active EoE (n = 102; mean age 7.1 years; 76% boys, 24% girls) were randomly assigned 2:2:1:1 to receive weight-tiered higher-exposure dupilumab, lower-exposure dupilumab or placebo for 16 weeks (part A), followed by a 36-week extension period active treatment phase in which eligible patients in each dupilumab group continued the same regimen and those in the placebo groups were assigned to either lower-exposure or higher-exposure dupilumab (part B).