The US Food and Drug Administration (FDA) has approved Dupilumab (Dupixent, Regeneron/Sanofi) for the treatment of eosinophilic esophagitis (EoE) in children aged 1 to 11 years and weighing ≥15 kg. It is the first and only medication approved for treating these patients.
The FDA had previously approved the medication in May 2022 for EoE in individuals aged 12 years and weighing ≥40 kg, as reported by Medscape Medical News.
EoE is a chronic inflammatory condition caused by type 2 inflammation that damages the esophagus and causes difficulty swallowing and eating.
Dupilumab is a monoclonal antibody that inhibits part of the inflammation pathway.
EoE KIDS Trial
The FDA approval of Dupilumab for younger children is based on the results of the randomized, double-blind, placebo-controlled Phase 3 EoE KIDS trial, which consisted of two parts.
Part A was a 16-week double-blind treatment phase that assessed the safety and efficacy of Dupilumab in a graded, weight-based dosing regimen.
After 16 weeks, 66% of children receiving a higher dose of Dupilumab in graded weight-based dosing regimens achieved histological disease remission (six or fewer eosinophils/high-power field), which was the primary endpoint, compared to only 3% of children receiving Dupilumab placebo.
Additionally, children treated with Dupilumab for 16 weeks experienced a greater decrease in the proportion of days with one or more signs of EoE according to the Pediatric EoE Sign/Symptom Questionnaire Caregiver Version (PESQ-C) compared to placebo.
Part B was a 36-week extended active treatment period in which eligible children from Part A continued to receive their dose in the Dupilumab group, and those in the placebo group in Part A switched to active treatment.
Histological remission persisted in week 52 for 53% of children treated with Dupilumab in Parts A and B. Histological remission was also achieved in week 52 for 53% of children who switched from placebo to Dupilumab in Part B.
The safety profile of Dupilumab observed in these children over 16 weeks was generally consistent with that observed in individuals aged 12 years and older with EoE over 24 weeks.
The most common adverse events (≥2%) observed more frequently with Dupilumab than with placebo were injection site reactions, upper respiratory tract infections, arthralgia, and herpes virus infections. In Part B of EoE KIDS, a case of helminth infection was reported in the Dupilumab arm.
Complete prescribing information is available online.