The European Commission has granted approval for Dupixent (dupilumab) for the treatment of eosinophilic esophagitis (EoE) in children aged 1 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 decision is based on data from the Phase 3 EoE KIDS trial, which demonstrated the drug's efficacy and safety in this age group.
Clinical Trial Data
The approval is rooted in the findings of the two-part EoE KIDS Phase 3 trial. In Part A, children receiving a higher, weight-based dose of Dupixent (n=37) were compared to those receiving a placebo (n=34) over 16 weeks. The primary endpoint was histological remission, defined as ≤6 eosinophils per high power field. Results showed that 68% of children on Dupixent achieved histological remission, compared to only 3% in the placebo group. This effect was sustained for up to one year in Part B of the trial.
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. The treatment also led to reductions in abnormal endoscopic findings and improvements in disease severity and extent, as measured microscopically. Caregiver-reported outcomes indicated a nominally significant improvement in the frequency and severity of EoE signs, along with a numerical reduction in days with at least one sign of EoE.
Safety Profile
The safety profile of Dupixent in the EoE KIDS trial was consistent with its established safety profile in adolescents and adults with EoE. Common adverse reactions included injection site reactions, conjunctivitis, arthralgia, oral herpes, and eosinophilia. In the 1 to 11-year-old group, adverse events more frequently observed with Dupixent included COVID-19, nausea, injection site pain, and headache. The long-term safety profile assessed in Part B of the trial mirrored the findings from Part A.
Addressing Unmet Needs in EoE
EoE is a chronic, progressive disease driven by type 2 inflammation, leading to esophageal damage and impaired function. Symptoms, often mistaken for other conditions, can severely impact a child’s ability to eat, causing vomiting, abdominal pain, difficulty swallowing, decreased appetite, and challenges in thriving. Continuous management is crucial to mitigate complications and disease progression.
According to Houman Ashrafian, M.D., Ph.D., Executive Vice President, Head of Research and Development at Sanofi, up to half of all children in the EU with EoE remain uncontrolled despite existing standard of care treatments. This approval offers a new treatment option for pediatric patients who previously lacked specifically approved therapies, potentially improving their chances to thrive.
Dosing and Administration
Dupixent is administered via subcutaneous injection. For children aged 1 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. The injection is intended for use under the guidance of a healthcare professional and can be administered in a clinic or at home by a trained caregiver.
