RARE Daily

FDA Expands Use of Dupixent to Include Kids with EoE 1 Year and Older

January 26, 2024

Rare Daily Staff

The U.S. Food and Drug Administration has expanded the approval of Dupixent to include the treatment of pediatric patients aged 1 to 11 years with the rare and chronic inflammatory condition of the esophagus eosinophilic esophagitis.

Dupixent is now the first and only medicine approved in the United States specifically indicated to treat these patients. The approval expands the initial FDA approval for EoE in May 2022 for patients aged 12 years and older. The FDA evaluated Dupixent for this expanded indication under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions.

Eosinophilic esophagitis (EoE) is a progressive disease driven in part by type 2 inflammation that damages the esophagus and impairs its function. EoE can severely impact a child’s ability to eat, and they may experience heartburn, vomiting, abdominal discomfort, trouble swallowing, food refusal, and failure to thrive. These symptoms can adversely impact their growth and development. Continuous treatment of EoE may be needed to reduce the risk of complications and disease progression.

Dupixent was invented by Regeneron and jointly developed with Sanofi. It is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways. It is not an immunosuppressant. The Dupixent development program has shown significant clinical benefit and a decrease in type 2 inflammation in phase 3 trials, establishing that IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases.

Dupixent has received regulatory approvals in one or more countries around the world for use in certain patients with atopic dermatitis, asthma, CRSwNP, EoE, and prurigo nodularis in different age populations. Dupixent is currently approved for one or more of these indications in more than 60 countries, including in Europe, the United States, and Japan.

The FDA approval is based on data from the phase 3 EoE KIDS trial with two parts (Part A and Part B) evaluating the efficacy and safety of Dupixent in children aged 1 to 11 years with EoE. At 16 weeks, 66 percent of children who received higher dose Dupixent at tiered dosing regimens based on weight achieved histological disease remission, the primary endpoint, compared to 3 percent for placebo. Histological remission was sustained at week 52 with 53 percent of children treated with Dupixent in Parts A and B. Histological remission was also achieved at week 52 in 53 percent of children who switched to Dupixent from placebo in Part B.

In addition, a greater decrease in the proportion of days with one or more signs of EoE based on Pediatric EoE Sign/Symptom Questionnaire-caregiver version was observed in children treated with Dupixent at 16 weeks compared to placebo.

“Young children with eosinophilic esophagitis have significant unmet medical needs; despite existing treatment options, 40 percent of these children in the U.S. under the age of 12 continue to experience symptoms of this disease,” said Naimish Patel, head of global development, immunology and inflammation at Sanofi. “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.”

Photo: Naimish Patel, head of global development, immunology and inflammation at Sanofi

 

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