Key Takeaways
- Dupilumab (Dupixent) is the first FDA-approved treatment for children aged 1 to 11 years, weighing at least 15 kg, who have esophagitis eosinophilic (EoE).
- EoE is a chronic, progressive disease that can severely impact a child's ability to eat and can cause a variety of symptoms, including heartburn, trouble swallowing, and food refusal.
- In the phase 3 EoE KIDS trial, 66% of children receiving a higher dose of dupilumab achieved histological disease remission, the primary end point, compared with 3% on placebo.
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.
