The European Medicines Agency (EMA) has granted approval to Dupixent (dupilumab) for the treatment of eosinophilic esophagitis (EoE) in children aged 1 to 11 years, marking a significant advancement for young patients struggling with this chronic condition. The approval specifically targets 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 approval in the European Union for EoE in adults and adolescents, establishing it as the first and only medicine indicated for treating this young patient population. Dupixent has also received approval for this age group in the United States and Canada.
The approval is based on data from the two-part (Part A and B) EoE KIDS phase 3 study, which demonstrated that the response to Dupixent in children with EoE is similar to that observed in adult and adolescent populations. In Part A of the study, children receiving a higher, weight-based dose of Dupixent (n=37) experienced significantly improved outcomes compared to those receiving placebo (n=34) after 16 weeks. Specifically, 68% of children in the Dupixent group achieved histological disease remission (≤6 eosinophils/high power field), compared to only 3% in the placebo group (p<0.0001), meeting the primary endpoint.
Sustained Efficacy and Safety
The benefits observed in Part A of the study were sustained for up to one year in Part B. Key findings included an 86% reduction in peak esophageal intraepithelial eosinophil count from baseline in the Dupixent group, compared to a 21% increase in the placebo group. Furthermore, the Dupixent group showed reductions in abnormal endoscopic findings and disease severity, along with nominally significant improvements in the frequency and severity of EoE signs based on caregiver-reported outcomes.
The safety profile of Dupixent in the EoE KIDS study was generally consistent with its known safety profile in adolescents and adults with EoE. The most common adverse reactions associated with Dupixent overall include injection site reactions, conjunctivitis, arthralgia, and oral herpes. In patients aged 1 to 11 years, adverse events more commonly observed with Dupixent (≥10%) compared to placebo during Part A included COVID-19, nausea, injection site pain, and headache. The long-term safety profile evaluated in Part B was similar to that observed during Part A.
Impact on Patient Care
EoE is a chronic, progressive disease characterized by type-2 inflammation, which damages the esophagus and impairs its function. Diagnosis can be challenging, as symptoms often mimic other conditions, leading to delays in treatment. The condition can significantly impact a child's ability to eat, causing vomiting, abdominal pain, difficulty swallowing, decreased appetite, and challenges in thriving. Continuous management is often necessary to reduce the risk of complications and disease progression.
According to Houman Ashrafian, MD, PhD, Executive Vice President, Head of Research and Development at Sanofi, up to half of all children in the EU with eosinophilic esophagitis remain uncontrolled despite existing standard of care treatment options. As a result, many of these young patients struggle to maintain weight due to serious symptoms such as difficulty swallowing and vomiting. This approval provides an important new treatment for pediatric patients who were previously without options specifically approved for their disease.
Study Details
The EoE KIDS phase 3 study was a randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of Dupixent in children aged 1 to 11 years with EoE. Part A enrolled 71 patients and evaluated Dupixent at a weight-based dose regimen, compared to placebo, for 16 weeks. Part B was a 36-week extended active treatment period in which eligible children from Part A in the Dupixent group continued treatment, while those in the placebo group switched to Dupixent. Patients included in this trial were previously treated and did not respond to conventional medicinal therapies, including proton pump inhibitors and/or swallowed topical corticosteroids.
The primary endpoint was histologic remission at 16 weeks, and secondary endpoints included assessments of endoscopic and histopathologic measures of the severity of disease along with caregiver-reported clinical signs and symptoms of EoE. The 108-week open-label extension period (Part C) to evaluate longer-term outcomes was recently completed.
About Dupixent
Dupixent (dupilumab) is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. In patients aged 1 to 11 years with EoE, Dupixent is administered 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. Dupixent is intended for use under the guidance of a healthcare professional and can be given in a clinic or at home administered by a caregiver after training by a healthcare professional.
Dupixent has received regulatory approvals in more than 60 countries for various indications, including atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, EoE, prurigo nodularis, chronic spontaneous urticaria, and chronic obstructive pulmonary disease in different age populations. It is being jointly developed by Sanofi and Regeneron under a global collaboration agreement.
