The European Medicines Agency has granted approval to Dupixent (dupilumab) for the treatment of eosinophilic esophagitis (EoE) in children aged one to 11 years. This approval marks Dupixent as the first and only medicine available in the European Union for this young patient population, addressing a significant gap in treatment options.
The approval is specifically for children weighing at least 15 kg who are inadequately controlled by, intolerant to, or ineligible for conventional medicinal therapies. This decision expands Dupixent's existing EU approval for EoE in adults and adolescents.
Clinical Trial Data Supporting Approval
The approval is based on data from the two-part EoE KIDS phase 3 study. Part A of the study involved children aged one to 11 years, with a higher dose of Dupixent (n=37) administered based on a weight-based regimen, compared to placebo (n=34). After 16 weeks, 68% of children on Dupixent achieved histological disease remission (≤6 eosinophils/high power field) compared to 3% on placebo (p<0.0001), meeting the primary endpoint. These results were sustained for up to one year in Part B of the study.
Additional findings from Part A include:
- 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.
Safety Profile
The safety profile of Dupixent in the EoE KIDS study was consistent with its known safety profile in adolescents and adults with EoE. Common adverse reactions included injection site reactions, conjunctivitis, arthralgia, and oral herpes. In patients aged one to 11 years, adverse events more commonly observed with Dupixent (≥10%) compared to placebo during Part A were COVID-19, nausea, injection site pain, and headache.
Expert Commentary
"Up to half of all children in the EU with eosinophilic esophagitis remain uncontrolled despite existing standard of care treatment options, and, as a result, many of these young patients struggle to maintain weight due to serious symptoms such as difficulty swallowing and vomiting," said Houman Ashrafian, MD, PhD, Executive Vice President, Head of Research and Development, Sanofi. "This milestone provides an important new treatment for pediatric patients who were previously without options specifically approved for their disease. With this novel approach to addressing an underlying cause of eosinophilic esophagitis, Dupixent has the potential to give these young children a better chance to thrive."
About Eosinophilic Esophagitis (EoE)
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. Continuous management is often necessary to mitigate complications and disease progression.
Dosing and Administration
Dupixent is administered via subcutaneous injection. In children aged one to 11 years with EoE, the dosing regimen is either 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.
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. It is not an immunosuppressant.
