Dupixent Receives EU Approval as First Treatment for Pediatric Eosinophilic Esophagitis
Key Insights
Dupixent (dupilumab) gains EU approval as the first treatment for eosinophilic esophagitis (search) in children aged 1-11, marking a significant advancement in pediatric care.
Phase III trial data demonstrated that 68% of children treated with Dupixent achieved histological disease remission at 16 weeks, sustained for up to one year.
The approval addresses a critical unmet need, as up to half of EU children with eosinophilic esophagitis (search) remain uncontrolled with standard treatments.
The European Medicines Agency (EMA) has approved Dupixent (dupilumab) as the first treatment for eosinophilic esophagitis (search) in the EU for children aged 1 to 11 years, weighing at least 15kg, who are inadequately controlled by, intolerant to, or are not candidates for conventional medicinal therapy. This approval marks a significant advancement in treating this condition in young patients.
The authorization is based on Phase III clinical trial data, which demonstrated that 68 percent of children receiving Dupixent achieved histological disease remission at 16 weeks, with results maintained for up to one year. The Phase III trial also showed an 86 percent reduction in peak esophageal intraepithelial eosinophil count at 16 weeks in Part A of the study, among children given a higher, weight-based dose of Dupixent.
Clinical Significance
Eosinophilic esophagitis (search) is a chronic, immune-mediated inflammatory disease characterized by an accumulation of eosinophils in the esophagus, leading to tissue damage and difficulty swallowing. The condition can significantly impair quality of life, especially in children. According to Dr. Houman Ashrafian, PhD, Executive Vice President, Head of Research and Development, Sanofi, Dupixent's novel approach addresses an underlying cause of eosinophilic esophagitis, potentially giving young children a better chance to thrive.
Unmet Needs Addressed
Despite existing standard of care treatment options, up to half of all children in the EU with eosinophilic esophagitis (search) remain uncontrolled. This approval provides an important new treatment for pediatric patients who were previously without specifically approved options for their disease.
Trial Details
The Phase III trial evaluated the efficacy and safety of Dupixent in children with eosinophilic esophagitis (search). The primary endpoint was histological remission at 16 weeks, defined as a peak esophageal intraepithelial eosinophil count of ≤6 eosinophils/high-power field (eos/HPF). Secondary endpoints included changes in disease symptoms and quality of life. The findings from this study were published in The New England Journal of Medicine.