FDA Approves Dupilumab for Pediatric Patients with Eosinophilic Esophagitis

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The approval marks dupilumab as the first and only FDA-approved medicine to treat pediatric patients with eosinophilic esophagitis.

The FDA has approved dupilumab (Dupixent; Regeneron Pharmaceutics and Sanofi) to treat pediatric patients aged 1 to 11 years and weighing at least 15 kg with eosinophilic esophagitis (EoE). The approval marks the first FDA-approved medicine to treat this pediatric patient population with EoE, according to a press release.

This new approval is an expansion of the May 2022 approval of dupilumab for adults and children 12 years and older weighing at least 40 kg, noted the study authors.

Disorders of swallowing, dysphagia. Pharyngeal and esophageal dysphagia. Oral phase. The path of food, the act of swallowing. Head side view x-ray, From the mouth to the stomach. 3d rendering - Image credit: Naeblys | stock.adobe.com

Image credit: Naeblys | stock.adobe.com

“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, in a press release. “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.”

EoE is a progressive disease that is caused by type 2 inflammation that harms the esophagus and disrupts its functions, resulting in difficulty eating. Children with EoE could experience heartburn, vomiting, abdominal discomfort, trouble swallowing, food refusal, and failure to thrive, according to study authors. The symptoms have been shown to negatively impact the growth and development of children with EoE.

The exact number of children impacted by EoE is unknown, although approximately 21,000 United States children under the age of 12 are being treated with unapproved therapies, according to the press release. The study authors noted that dupilumab is a “fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant.”

The FDA approval was based on results from the randomized, double-blind, placebo-controlled phase 3 EoE KIDS trial. In the trial, parts A and B assessed the efficacy and safety of dupilumab in children 1 to 11 years old with EoE, compared with a placebo. Part A lasted 16 weeks and included 61 children, and part B extended for 36 weeks.

At 16 weeks, 66% of children who received a higher dose of dupilumab based on their weight attained histological disease remission, compared with 3% in the placebo group. Remission was also achieved at 52 weeks among 53% of children who switched to dupilumab in part B after receiving the placebo.

The study authors noted that safety profile displayed similar results in children aged 1 to 11 at 16 weeks, as it did for adults and pediatric patients aged 12 and older at 24 weeks.

The most common adverse events among children that received dupilumab included injection site reactions, upper respiratory tract infections, arthralgia, and herpes viral infections.

“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, in a press release. “With this approval, Dupixent becomes the first and only treatment option for EoE patients aged 1 year and older, weighing at least 15 kg. By targeting the underlying type 2 inflammation that contributes to this disease, Dupixent has the potential to transform the standard of care for these children as it has for adults and adolescents with EoE.”

Reference

Press Release: Dupixent® FDA approved as first and only treatment indicated for children aged 1 year and older with eosinophilic esophagitis (EoE). Globe News Wire. News release. January 25, 2024. Accessed January 25, 2024. https://www.globenewswire.com/news-release/2024/01/25/2817342/0/en/Press-Release-Dupixent-FDA-approved-as-first-and-only-treatment-indicated-for-children-aged-1-year-and-older-with-eosinophilic-esophagitis-EoE.html.

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