EU Approves Dupixent for Young Children with Eosinophilic Esophagitis

PARIS, France and TARRYTOWN, NY, USA — November 6, 2024

The European Medicines Agency has granted approval for Dupixent (dupilumab) to be used in treating eosinophilic esophagitis (EoE) in children starting from the age of one year. This approval is specific to children aged one to 11 years who weigh at least 15 kg and who have not responded adequately to conventional medicinal therapies, are intolerant to such treatments, or are not considered suitable candidates for them. This new approval extends the initial authorization that covered adults and adolescents within the European Union (EU), making Dupixent the inaugural medication approved for this young demographic in the EU. It is also approved for use in this age group in the US and Canada.

Roberta Giodice, President of ESEO Italia, expressed the significance of this development. She noted that young children with EoE face a lifelong challenge that impacts their eating abilities. Parents have often resorted to restrictive diets that do not directly address the disease, potentially affecting children's growth during critical development periods. The continued research and new treatment options provide hope for improved care quality for these young patients.

Houman Ashrafian, MD, PhD, Executive Vice President and Head of Research and Development at Sanofi, highlighted that despite existing treatments, up to half of the children with EoE in the EU remain uncontrolled. The new approval offers an important treatment option for pediatric patients who previously had no specifically approved therapy for their condition. Dupixent's novel approach targets an underlying cause of EoE, potentially improving the lives of affected children.

The approval is based on the EoE KIDS phase 3 study, which was conducted in two parts (Part A and B). This study involved children aged one to 11 years and established a connection showing that the response to Dupixent in children is consistent with that seen in adults and adolescents. Part A of the study indicated that children who received a higher dose of Dupixent based on their weight experienced significant outcomes compared to those who received a placebo over 16 weeks. The safety results were generally consistent with the known safety profile of Dupixent. Common adverse reactions included injection site reactions, conjunctivitis, allergic conjunctivitis, arthralgia, oral herpes, and eosinophilia. Additionally, injection site bruising was also reported. Common adverse events in children aged one to 11 years included COVID-19, nausea, injection site pain, and headaches.

George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President, and Chief Scientific Officer at Regeneron, emphasized that EoE poses a unique challenge for young children, affecting their basic ability to eat during a critical growth period. The new approval brings the established efficacy and safety profile of Dupixent to this vulnerable population, potentially transforming the standard of care for young patients.

EoE is a chronic and progressive disease linked to type-2 inflammation that damages the esophagus and impairs its function. Symptoms can be mistaken for other conditions, leading to delays in diagnosis. EoE can severely affect a child's ability to eat, causing symptoms like vomiting, abdominal pain, difficulty swallowing, decreased appetite, and growth challenges. Continuous management is necessary to reduce the risk of complications and disease progression.

The EoE KIDS phase 3 study was a randomized, double-blind, placebo-controlled trial evaluating Dupixent's efficacy and safety in children aged one to 11 years with EoE. Part A involved 71 patients and assessed Dupixent at a weight-based dose regimen compared to placebo over 16 weeks. Part B extended the active treatment period for 36 weeks. Patients in this trial had previously not responded to conventional therapies, including proton pump inhibitors and swallowed topical corticosteroids. The primary endpoint was histologic remission at 16 weeks, with secondary endpoints assessing disease severity and caregiver-reported clinical signs and symptoms. The study's results were published in The New England Journal of Medicine.

Dupixent, administered via subcutaneous injection, is given every other week or weekly based on the child's weight. It is a fully human monoclonal antibody that inhibits interleukin-4 (IL4) and interleukin-13 (IL13) pathways and is not an immunosuppressant. Its development program has demonstrated significant clinical benefits and reduced type-2 inflammation in multiple studies. Dupixent has received regulatory approvals in over 60 countries for various indications, including atopic dermatitis, asthma, and EoE, among others.