FDA puts Dupixent in priority review for treatment of eosinophilic esophagitis in children younger than 12September 29, 2023
Regeneron Pharmaceuticals and Sanofi have received U.S. Food and Drug Administration (FDA) acceptance for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent (dupilumab) to treat children aged 1 to 11 years with eosinophilic esophagitis (EoE).
If approved, Dupixent would be the first and only treatment indicated in the U.S. for children aged 1- 11 with eosinophilic esophagitis, a disease driven by type 2 inflammation that impacts the ability to eat.
The target action date for the FDA decision on this is January 31, 2024.
Children suffering from EoE often face common symptoms including heartburn, acid reflux, vomiting, abdominal discomfort, trouble swallowing, food refusal and failure to thrive. These symptoms can impact their growth and development at such a pivotal age. The sBLA is supported by data from the Phase 3 EoE KIDS trial (Parts A and B) evaluating the efficacy and safety of Dupixent in children aged 1 to 11 with EoE.
In Part A, the primary endpoint was met for the proportion of patients achieving histological disease remission (defined as peak esophageal intraepithelial eosinophil count of ≤6 eosinophils [eos]/high power field [hpf]) at 16 weeks for tiered dosing regimens based on body weight, compared to placebo. Part B was an active treatment extension period evaluating Dupixent for an additional 36 weeks and showed Dupixent maintained histologic remission for 52 weeks, a secondary endpoint. Dupixent also led to increases in body weight for age percentile, which was evaluated as an exploratory endpoint in Part A and a secondary endpoint in Part B.
About Eosinophilic Esophagitis
EoE is a chronic, progressive disease driven by type 2 inflammation that damages the esophagus and prevents it from working properly. In children, common symptoms of EoE include heart burn, vomiting, abdominal discomfort, trouble swallowing, food refusal and failure to thrive. These symptoms can impact growth and development and can cause food-related fear and anxiety, which can persist through adulthood. Dietary adjustments, including the elimination of many foods, are the standard treatment for EoE, as well as the use of treatments not approved for the disease. These include proton pump inhibitors, swallowed topical corticosteroids, or in severe cases, a feeding tube, which may be used to ensure proper caloric intake and weight gain. Continuous treatment of EoE may be needed to reduce the risk of complications and disease recurrence.
Dupixent is a fully human monoclonal antibody that inhibits the signaling of the IL-4 and IL-13 pathways and 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. These diseases include approved indications for Dupixent, such as atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis (CRSwNP), prurigo nodularis and EoE.