Dupixent Approved in EU for Young Children with Eosinophilic Esophagitis
Key Insights
Dupixent (dupilumab) has been approved in the EU as the first and only medicine for children aged 1 to 11 years with eosinophilic esophagitis (search) (EoE (search)).
In a Phase 3 trial, 68% of children achieved histological disease remission with Dupixent compared to 3% with placebo, sustained for up to one year.
The study also demonstrated significant reductions in esophageal eosinophil count and improvements in endoscopic findings and caregiver-reported outcomes.
Dupixent (dupilumab) has received approval in the European Union as the first and only treatment specifically for children aged 1 to 11 years suffering from eosinophilic esophagitis (search) (EoE (search)). This approval marks a significant advancement in the management of EoE, a chronic, progressive disease characterized by type-2 inflammation that damages the esophagus and impairs its function. The decision is based on the positive results from the Phase 3 EoE KIDS study, which demonstrated significant efficacy and a favorable safety profile in this vulnerable patient population.
The EoE (search) KIDS study was a randomized, double-blind, placebo-controlled trial that evaluated the efficacy and safety of Dupixent in children aged 1 to 11 years with EoE. The study included patients who had previously been treated but did not respond to conventional therapies such as proton pump inhibitors and/or swallowed topical corticosteroids. Part A of the study 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.
Key Findings from the EoE KIDS Study
The primary endpoint of the study was histologic remission at 16 weeks, defined as ≤6 eosinophils/high power field. Key findings include:
- Histologic Remission: 68% of patients treated with Dupixent achieved histological disease remission compared to only 3% in the placebo group (p<0.0001). This effect was sustained for up to one year in Part B of the study.
- Esophageal Eosinophil Count: An 86% reduction in peak esophageal intraepithelial eosinophil count from baseline was observed in the Dupixent group, compared to a 21% increase in the placebo group.
- Endoscopic Improvements: Reductions in abnormal endoscopic findings and disease severity and extent were noted at the microscopic level.
- Symptom Improvement: Nominally significant improvement in the frequency and severity of EoE signs, and numerical reduction in days with at least one sign of EoE, were reported by caregivers.
Safety Profile
The safety profile of Dupixent in the EoE (search) KIDS study was generally consistent with its known safety profile in adolescents and adults with EoE. Common adverse reactions included injection site reactions, conjunctivitis, arthralgia, oral herpes, and eosinophilia. 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. The long-term safety profile evaluated in Part B was similar to that observed during Part A.
Implications for EoE Treatment
EoE (search) is a chronic condition that can severely impact a child’s ability to eat, causing vomiting, abdominal pain, difficulty swallowing, decreased appetite, and challenges in thriving. Continuous management is often needed to reduce the risk of complications and disease progression. This approval offers a much-needed therapeutic option for young children with EoE who have had limited treatment choices.
George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President, and Chief Scientific Officer at Regeneron, stated, “Eosinophilic esophagitis (search) presents a unique challenge in young children, who struggle with their basic ability to eat during a time in their lives where proper nutrition is essential for growth and development. 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 (search) patients and has the potential to transform the standard of care for children with EoE who previously had no therapies specifically approved for them.”
Dosing and Administration
Dupixent is administered via subcutaneous injection. In patients aged one to 11 years with EoE (search), it is given 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.
About Dupixent
Dupixent (dupilumab) is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (search) (IL-4 (search)) and interleukin-13 (search) (IL-13 (search)) pathways, key drivers of type-2 inflammation. It is not an immunosuppressant. Dupixent has received regulatory approvals in more than 60 countries for various indications, including atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, EoE (search), prurigo nodularis, chronic spontaneous urticaria, and chronic obstructive pulmonary disease in different age populations. Over 1,000,000 patients are being treated with Dupixent globally.
Dupixent is being jointly developed by Sanofi and Regeneron under a global collaboration agreement.