FDA Approves Dupilumab to Treat EoE for Children Under 12 Years

The FDA has expanded the indication for dupilumab (Dupixent) for the treatment of eosinophilic esophagitis (EoE) in pediatric patients aged 1 to 11 years who weigh at least 15 kg. This approval marks the first treatment of EoE for this age group.¹

Dupilumab was first approved to treat EoE in May 2022 for adults and children 12 years and older.²

EoE is a chronic, progressive disease thought to be responsible for damaging the esophagus, and it can severely impact a child’s ability to eat. Additional symptoms of EoE include heartburn, vomiting, abdominal discomfort, trouble swallowing, food refusal, and failure to thrive.

“Young children are some of the most vulnerable patients with eosinophilic esophagitis, or EoE, as this debilitating and progressive disease threatens their basic ability to eat. Until today, these children had no approved treatment options specifically for EoE, leaving many with unapproved medicines that failed to target the root cause of their disease,” said George D. Yancopoulos, MD, PhD, board cochair, president, and chief scientific officer at Regeneron.

Although there are approximately 21,000 children 11 years and younger being treated for EoE, the actual prevalence may be higher as the symptoms are often mistaken for other conditions.

The FDA approval is based on data from the phase 3 EoE KIDS trial, which included 2 parts to evaluate the safety and efficacy of dupilumab in children under the age of 12. At baseline in the randomized, double-blind, placebo-controlled trial, 97% of patients had at least 1 coexisting type 2 inflammatory disease. The first part of the trial (Part A) was a 16-week double-blind treatment period with 61 patients. The second part (Part B) was a 36-week extended active treatment period for 47 children from Part A. Those on dupilumab in Part A remained on it, while children on placebo in Part A were switched to dupilumab.

At the end of Part A, 66% of the 32 children receiving a higher dose of dupilumab based on weight achieved histological disease remission, the primary end point, compared with only 3% on placebo. More than half (53%) of children treated in Parts A and B who achieved histological remission sustained it at week 52. In addition, 53% of the children who were on placebo in Part A and switched to dupilumab in Part B had achieved a histological remission at week 52.

Based on responses to the Pediatric EoE Sign/Symptom Questionnaire-caregiver version, children treated with dupilumab had a greater decrease in the proportion of days with 1 or more signs of EoE at week 16 compared with children in the placebo group.

The safety profile for Part A was similar to that seen through 24 weeks in patients older than 12 years. The most common adverse events (> 2%) for dupilumab were injection site reactions, upper respiratory tract infections, arthralgia, and herpes viral infections.

“Young children with eosinophilic esophagitis have significant unmet medical needs; despite existing treatment options, 40% of these children in the US under the age of 12 continue to experience symptoms of this disease,” said Naimish Patel, MD, Head of Global Development, Immunology and Inflammation at Sanofi.¹ “Today’s approval underscores our commitment to bringing therapies to young patients with unmet needs and also brings hope to these patients who are at a critical age where struggling to eat and maintain weight directly impacts their overall nutritional intake and development.”

References

1. Dupixent (dupilumab) FDA approved as first and only treatment indicated for children aged 1 year and older with eosinophilic esophagitis (EoE). News release. Regeneron. January 25, 2024. Accessed January 25, 2024. https://investor.regeneron.com/news-releases/news-release-details/dupixentr-dupilumab-fda-approved-first-and-only-treatment

2. Inserro A. FDA approves dupilumab as first therapy for eosinophilic esophagitis. The American Journal of Managed Care^®. May 20, 2022. Accessed January 25, 2024. https://www.ajmc.com/view/fda-approves-dupilumab-as-first-therapy-for-eosinophilic-esophagitis