Dupixent Phase 3 Data in Children with Eosinophilic Esophagitis Published in NEJM

The New England Journal of Medicine recently published promising phase 3 study results for Dupixent (dupilumab) in treating eosinophilic esophagitis (EoE) in children aged one to 11 years. This study revealed that a significant number of children receiving weight-tiered higher doses of Dupixent exhibited marked improvements in several key measures of EoE, compared to those given a placebo over a 16-week period.

EoE is a chronic, progressive condition linked to type-2 inflammation, which is believed to damage the esophagus and hinder its function. Diagnosing EoE poses challenges, as its symptoms can be easily confused with other ailments, leading to delays. The disease can severely impair a child’s ability to eat, causing abdominal pain, swallowing difficulties, heartburn, vomiting, and failure to thrive. Continuous management is often required to prevent complications and further progression of the disease.

Dr. Mirna Chehade, a principal investigator of the study at the Mount Sinai Center for Eosinophilic Disorders, emphasized the significance of these findings, noting that they could potentially change the standard of care for young children suffering from EoE. The study demonstrated that weight-tiered higher doses of Dupixent significantly improved multiple histologic, endoscopic, and cellular measures of EoE in children as young as one year old, with sustained benefits extending up to a year. These results align with earlier positive outcomes observed in older patients, reinforcing the role of IL4 and IL13 as key drivers of type 2 inflammation in EoE.

The study, a randomized, double-blind, placebo-controlled trial, evaluated the efficacy and safety of Dupixent in 102 children aged one to 11 years. Participants were assigned to either a weight-tiered higher dose or lower dose regimen of Dupixent, or a placebo, for 16 weeks (part A). Part B consisted of a 36-week extended treatment period, where children initially on Dupixent maintained their dosage, while those on placebo switched to Dupixent. The primary endpoint was histologic remission at 16 weeks, with secondary endpoints assessing the severity of disease through endoscopic and histopathologic measures, along with clinical signs and symptoms of EoE. Change in body weight for age percentile was also evaluated as an exploratory endpoint in part A and as a secondary endpoint in part B.

The safety outcomes were generally in line with the known safety profile of Dupixent in adolescents and adults with EoE. Common adverse events (≥10%) in the study included COVID-19, nausea, injection site pain, and headache. The long-term safety profile for children aged one to 11 years in part B mirrored that observed in part A, with one case of helminth infection reported with Dupixent.

For U.S. patients with EoE weighing at least 15 kg, the FDA-approved dosage for Dupixent is 200mg or 300mg every other week, or 300mg weekly, based on weight.

Dupixent is a fully human monoclonal antibody that inhibits the signaling pathways of interleukin-4 (IL4) and interleukin-13 (IL13), crucial drivers of type 2 inflammation. The Dupixent development program has demonstrated significant clinical benefits and a reduction in type 2 inflammation across multiple phase 3 studies. Dupixent has received regulatory approval in over 60 countries for various indications, including atopic dermatitis, asthma, and chronic rhinosinusitis with nasal polyposis, among others. More than 850,000 patients globally are currently being treated with Dupixent. 

Dupilumab is jointly developed by Sanofi and Regeneron under a global collaboration agreement and has been studied in over 60 clinical trials involving more than 10,000 patients. Beyond the approved indications, ongoing phase 3 studies are exploring Dupixent's efficacy in additional type 2 inflammation-driven diseases, including chronic spontaneous urticaria and chronic obstructive pulmonary disease, among others.

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