The European Medicines Agency has approved Dupixent (dupilumab) for the treatment of eosinophilic esophagitis (EoE) in children aged 1 to 11 years, marking it as the first and only approved medication for this age group in the EU. This approval addresses a critical unmet need for young children who are inadequately controlled by conventional therapies, are intolerant to them, or are not candidates for such treatments.
The approval is based on data from the Phase 3 EoE KIDS study, a two-part trial that demonstrated the efficacy and safety of Dupixent in this young population. Part A of the study involved 71 children aged 1 to 11 years, with 37 receiving a weight-based higher dose of Dupixent and 34 receiving a placebo. The primary endpoint was histological remission at 16 weeks, defined as ≤6 eosinophils/high power field.
Key Findings from the EoE KIDS Study
At 16 weeks, 68% of children treated with Dupixent achieved histological disease remission compared to only 3% in the placebo group (p<0.0001). These results were sustained for up to one year in Part B of the study. Additional benefits observed in the Dupixent group included:
- An 86% reduction in peak esophageal intraepithelial eosinophil count from baseline, compared to a 21% increase in the placebo group.
- Reductions in abnormal endoscopic findings and disease severity.
- Nominally significant improvement in the frequency and severity of EoE signs based on caregiver-reported outcomes.
Clinical Significance
EoE is a chronic, progressive disease driven by type-2 inflammation, leading to esophageal damage and impaired function. Symptoms such as vomiting, abdominal pain, difficulty swallowing, and decreased appetite can severely impact a child's ability to eat and thrive. Up to half of all children in the EU with EoE remain uncontrolled despite existing standard of care treatment options.
"Young children with eosinophilic esophagitis are at the beginning of their life-long journey with a disease that challenges their ability to eat," said Roberta Giodice, President of ESEO Italia. "Parents of these children have often relied on restrictive diets that do not specifically address the disease and can stunt their growth at a critical time in development that could impact them for years to come. We are pleased that research continues and offers new treatment options to improve the quality of their care."
Safety Profile
The safety profile of Dupixent in the EoE KIDS study was consistent with previous findings in adolescents and adults with EoE. Common adverse reactions included injection site reactions, conjunctivitis, arthralgia, and oral herpes. In patients aged 1 to 11 years, adverse events more commonly observed with Dupixent were COVID-19, nausea, injection site pain, and headache.
Mechanism of Action
Dupixent is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways, key drivers of type-2 inflammation. By blocking these pathways, Dupixent helps to reduce inflammation in the esophagus and alleviate the symptoms of EoE.
"This approval will bring the proven efficacy and demonstrated safety profile of Dupixent to this vulnerable, young population that has already been established in older EoE patients and has the potential to transform the standard of care for children with EoE who previously had no therapies specifically approved for them,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President, and Chief Scientific Officer at Regeneron.
Dosing and Administration
In patients aged 1 to 11 years with EoE, Dupixent is administered via subcutaneous injection every other week (200 mg for children ≥15 to <30 kg, 300 mg for children ≥30 to <40 kg) or every week (300 mg for children ≥40 kg), based on weight. It is intended for use under the guidance of a healthcare professional and can be administered in a clinic or at home by a caregiver after proper training.
