FDA Expands Indication for Dupixent in Eosinophilic Esophagitis to Include Children Under 12 Years

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Approval based on findings from the Phase III EoE KIDS trial, in which 66% of patients administered a higher dose of Dupixent based on weight achieved histological disease remission.

Image credit: Yurii Kibalnik | stock.adobe.com

Image credit: Yurii Kibalnik | stock.adobe.com

The FDA has approved an expanded indication for Regeneron Pharmaceuticals’ and Sanofi’s Dupixent (dupilumab) to treat eosinophilic esophagitis (EoE) in patients aged 1 to 11 years weighing at least 33 lbs. The regulatory action makes Dupixent the first and only FDA-approved medication for EoE specifically in this patient population. The FDA approved the drug in May 2022 to treat EoE in patients aged 12 years and older, weighing at least 88 lbs.

“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 Regeneron board co-chair, president, and chief scientific officer 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 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.”

Dupixent is an interleukin-4 receptor alpha antagonist previously approved to treat EoE in patients aged 12 years and older weighing at least 88 lbs.; for adults with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable; asthma; chronic rhinosinusitis with nasal polyposis; and prurigo nodularis.

EoE is a chronic, progressive condition suggested to be responsible for causing damage to the esophagus, severely affecting a patient’s ability to eat. Additional symptoms of the disease include heartburn, vomiting, abdominal discomfort, trouble swallowing, food refusal, and failure to thrive. Approximately 21,000 US children under 12 years of age are currently being administered unapproved therapies for EoE, according to Regeneron.

The approval of the expanded indication for Dupixent was based on findings from the randomized, double-blind, placebo-controlled Phase III EoE KIDS trial. The trial included separate parts that analyzed the safety and efficacy of Dupixent in patients under 12 years of age.

At baseline, 97% of patients had at least one coexisting type 2 inflammatory disease. Part A of the trial was comprised of a 16-week double-blind period with 61 patients. Part B was comprised of a 36-week extended active treatment period for 47 children from Part A. Patients on Dupixent from Part A remained on the study treatment, whereas those administered placebo in Part A were switched to Dupixent.

Among those in Part A, 66% of 32 patients administered a higher dose of Dupixent based on weight achieved histological disease remission compared with 3% of patients in the placebo cohort, which was the trial’s primary endpoint. Among those treated in parts A and B who achieved histological remission, 53% sustained it at week 52, whereas 53% of patients in the placebo cohort in Part A who switched to Dupixent in Part B achieved a histological remission at week 52.

The safety profile in Part A of the trial was similar to what was observed through 24 weeks in patients older than 12 years of age. The most common adverse events (≥ 2%) associated with treatment 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, 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.”

Reference

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

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