REGN - Press Release: Dupixent positive phase 3 data in children one to 11 years of age with eosinophilic esophagitis published in NEJM | Benzinga
Dupixent positive phase 3 data in children one to 11 years of age with eosinophilic esophagitis published in NEJM
- Majority of patients in this age group with eosinophilic esophagitis receiving Dupixent achieved histologic remission, with improvements sustained up to one year
- Dupixent is the first-and-only medicine indicated for eosinophilic esophagitis in the US for this age group
Paris and Tarrytown, NY, June 26, 2024. The New England Journal of Medicine has published results from a positive phase 3 study of Dupixent (dupilumab) in children aged one to 11 years with eosinophilic esophagitis (EoE). The study showed a greater proportion of those receiving weight-tiered higher dose Dupixent experienced significant improvements in many key disease measures of EoE, compared to placebo at week 16. Data from the study were the basis for the US Food and Drug Administration Priority Review and approval of Dupixent in children aged one to 11 years with EoE weighing at least 15 kg, as well as for the regulatory submission that is currently under review by the European Medicines Agency for this age group.
EoE is a chronic, progressive disease associated with type-2 inflammation that is thought to be responsible for damaging the esophagus and impairing its function. Diagnosis is difficult, as symptoms can be mistaken for other conditions, and there are delays in diagnosis. EoE can severely impact a child's ability to eat and may also cause abdominal pain, trouble swallowing, heartburn, vomiting and failure to thrive. Continuous management of EoE may be needed to reduce the risk of complications and disease progression.
Mirna Chehade, M.D., MPH
Mount Sinai Center for Eosinophilic Disorders, Icahn School of Medicine at Mount Sinai, New York, NY, and principal investigator of the study
"The NEJM publication of these phase 3 dupilumab results is a testament to the importance of these data and potential for dupilumab to change the standard of care for many young children living with eosinophilic esophagitis. These children commonly experience feeding difficulties, food refusal and failure to thrive during a critical time of their growth and development. These data showed weight-tiered higher dose dupilumab significantly improved key eosinophilic esophagitis histologic, endoscopic, and cellular measures in children as young as 1 year old with sustained results for up to one year. These results reinforce the positive results seen in older patients with eosinophilic esophagitis and strengthen our understanding of IL4 and IL13 as key drivers of the type 2 inflammation underlying this disease."
As published, a significantly greater proportion of children receiving either a weight-tiered higher or lower dose regimen of Dupixent achieved histologic remission at week 16 in part A of the study, compared with placebo. Additionally, those treated with higher dose Dupixent experienced significant improvements in disease severity assessed by endoscopic measures, with improvements sustained for up to one year. Those receiving lower dose Dupixent experienced improvements that were either comparable or numerically lower than the higher dose group. Dupixent also led to numerical improvement in body weight for age percentile by week 16 and sustained to one year, which was evaluated as an exploratory endpoint in part A and a secondary endpoint in part B.
Safety results were generally consistent with the known safety profile of Dupixent in adolescents and adults with EoE. Adverse events more commonly observed with Dupixent (?10%) in either weight-based dosing regimen versus placebo in the study were COVID-19, nausea, injection site pain and headache during part A. The long-term safety profile of Dupixent in children aged one to 11 years through part B was similar to that observed during part A. In part B, one case of helminth infection was reported with Dupixent.
For patients in the US 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.
Dr. Mirna Chehade has served as a paid consultant for Sanofi and Regeneron and has received research grant funding from Regeneron.
About the Dupixent pediatric eosinophilic esophagitis study
The phase 3 randomized, double-blind, placebo-controlled study evaluated the efficacy and safety of Dupixent in children aged one to 11 years with EoE. Part A enrolled 102 patients and evaluated Dupixent at a weight-tiered higher dose or lower 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 maintained their weight-tiered higher or lower dose level, while those in the placebo group switched to weight-tiered higher or lower dose Dupixent.
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 clinical signs and symptoms of EoE. Change in body weight for age percentile was evaluated as an exploratory endpoint in part A and as a secondary endpoint in part B. The study is ongoing with a 108-week open-label extension period (part C) to evaluate longer-term ...