Long Time Coming: Pediatric Gastroenterologists React to New Dupilumab EoE Approval

On January 25, dupilumab (Dupixent) became the first and only treatment approved by the US Food and Drug Administration (FDA) for eosinophilic esophagitis (EoE) in pediatric patients aged 1-11 years weighing ≥15 kg.¹

The FDA approval for this indication came a week ahead of the anticipated target action date of January 31, with many pediatric gastroenterologists in agreement that the early decision for this long-awaited treatment option was a major win for a previously untreated patient population.^1,2

“For many, many years now, there have not been many treatment options for our young pediatric patients with EoE. Essentially, it was PPI, swallowed steroids, food elimination,” Riha Bhatt, MD, assistant professor of pediatrics, pediatric gastroenterology, hepatology, and nutrition at Vanderbilt University Medical Center, explained in an interview with HCPLive. “You're constantly doing, to be honest, what felt like band-aids. The approval for dupilumab for our teenagers, and now for our kids above 1 year of age, has been a huge development.”

Dupilumab was initially approved for EoE in patients ≥12 years of age weighing ≥40 kg in May 2022. The expanded indication in this younger patient population was granted following the FDA’s acceptance of Regeneron Pharmaceuticals’ supplemental Biologics License Application (sBLA) for Priority Review on September 26, 2023.^2,3

Victoria Baez, MD, assistant professor of pediatrics in the division of pediatric gastroenterology at the Icahn School of Medicine at Mount Sinai and Mount Sinai Kravis Children’s Hospital, explained that she expected dupilumab to be approved in this patient population and has been anticipating this decision. “I’ve probably been telling my patients for the past 5 years that it’s coming,” Baez told HCPLive. “This definitely becomes another option and an option that was needed for a long time.”

Prior to dupilumab’s approval, clinicians relied heavily on off-label prescriptions to treat EoE in younger children. Elizabeth Spencer, MD, assistant professor of pediatrics in the division of pediatric gastroenterology at Icahn School of Medicine at Mount Sinai and Mount Sinai Kravis Children’s Hospital, explained that a lot of EoE patients have comorbid atopic dermatitis, a condition for which dupilumab was already approved in this age range.

Despite clinicians’ efforts to administer dupilumab for both conditions, Spencer acknowledged that EoE-specific dosing remained an issue until its FDA approval for this indication, which provided the much sought-after information about optimal dosing. Indeed, results from the phase 3 EoE KIDS trial supporting the FDA decision showed more patients who received higher dose dupilumab at tiered dosing regimens based on weight achieved histological disease remission compared to those who received placebo (66% vs 3%, respectively).¹

“This really provides us with dosing information, which is very important in pediatrics, especially in a disease that causes growth issues, and then actually helps kids grow. So, we really want to make sure that someone is dosed appropriately,” Spencer explained.

Now, clinicians are tasked with deciding their priority of use and who will benefit most from the first-of-its-kind treatment option.

“I think priority will come for those who are refractory, who standard steroids or food elimination diet doesn't help, or it is truly recurrent in a very frequent way,” Bhatt said, also citing its use in patients who have other atopic conditions that can be treated with dupilumab.

Still, an unmet need remains in patients ≤1 year of age for whom dupilumab is not yet approved. “We're sort of holding our breath as to if we'll be able to use it in the EOE children as well,” Spencer said, hopeful for an eventual FDA decision and information about dosing in this patient population.

References:

1. ^{Fitch, J. FDA approves dupilumab for eosinophilic esophagitis in children aged 1 to 11 years. Contemporary Pediatrics. January 25, 2024. Accessed January 31, 2024. https://www.contemporarypediatrics.com/view/fda-approves-dupilumab-for-eosinophilic-esophagitis-in-children-aged-1-to-11-years}
2. ^{Brooks, A. FDA Accepts Priority Review Application for Dupilumab for Pediatric Eosinophilic Esophagitis. HCPLive. September 26, 2023. Accessed January 31, 2024. https://www.hcplive.com/view/fda-accepts-priority-review-application-for-dupilumab-for-pediatric-eosinophilic-esophagitis}
3. ^{Butera, A. FDA Approves Dupilumab for Eosinophilic Esophagitis. HCPLive. May 20, 2022. Accessed January 31, 2024. https://www.hcplive.com/view/fda-approves-dupilumab-for-eosinophilic-esophagitis}