FDA Accepts Dupilumab for Priority Review in Children Under 12 With EoE

A supplemental Biologics License Application (sBLA) for dupilumab (Dupixent) for the treatment of children from ages 1 to 11 with eosinophilic esophagitis (EoE) was accepted by the FDA for priority review. Regeneron and Sanofi, the developers of dupilumab, announced the sBLA's acceptance in a statement Wednesday.

The FDA has set a target action date of January 31, 2024.

FDA | Photo credit: Araki Illustrations - stock.adobe.com

If the sBLA is approved, dupilumab would be the first and sole treatment in the US designated for children under the age of 12 who have EoE. As data currently estimates, there are approximately 21,000 children under 12 undergoing EoE treatment in the US. If approved, dupilumab would provide a valuable, alternative option for those in this age range whose disease is not as well managed by other forms of treatment.

EoE is a chronic disease that worsens over time. In cases of EoE, type 2 inflammation affects the esophagus and its ability to function. As symptoms progress, it can drastically alter someone’s eating habits. Children can experience issues with heart burn, swallowing, vomiting, abdominal discomfort, and find themselves refusing food. Dupilumab works by inhibiting the signals within the interleukin (IL)-4 and IL-13 pathways.

The sBLA is supported by parts A and B of the phase 3 EoE KIDS trial (NCT04394351), in which children between the ages of 1 and 11 were split into three groups and administered either a lower dose of dupilumab, higher dose, or placebo. Over 50% of children demonstrated significant histological disease remission at 16 weeks.²

Results from the higher dose group also indicated:

• An 86% reduced peak esophageal intraepithelial eosinophil level compared with their baseline vs a 21% increase in the placebo group (P = .0001)
• Disease severity and extent reductions of 0.88 and 0.84 from baseline vs 0.02 and 0.05 in the placebo group (P = .0001)
• A reduction in abnormal endoscopic findings of 3.5 points from baseline vs a 0.3-point increase for the placebo group

“The efficacy of [dupilumab] demonstrates that, in this age group, as in adults, IL-4 and IL-13 are key drivers of the type 2 inflammation underlying this debilitating disease. Eosinophilic esophagitis can turn the basic and life-sustaining act of eating into a painful experience at a point in children’s lives when proper nutrition and achieving a healthy weight is critical to ensuring they grow and thrive," George D. Yancopoulos, MD, PhD, president and chief scientific officer at Regeneron, stated in a press release.² "The positive results from this Phase 3 pediatric trial show [dupilumab] has the potential to improve signs of eosinophilic esophagitis and support healthy weight gain in children from their first birthday.”

Dupilumab is already FDA approved for the treatment of adult patients with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when the use of such therapies is not recommended.³ Ongoing studies are assessing dupilumab in a wider range of diseases driven by type 2 inflammation or other allergic processes.¹

References

1. Dupixent (dupilumab) sBLA for treatment of eosinophilic esophagitis in children aged 1 to 11 accepted for priority review. Sanofi. September 26, 2023. Accessed September 27, 2023. https://www.news.sanofi.us/2023-09-26-Dupixent-R-dupilumab-sBLA-for-treatment-of-eosinophilic-esophagitis-in-children-aged-1-to-11-accepted-for-Priority-Review

2. Press release: Dupixent (dupilumab) phase 3 trial shows positive results in children 1 to 11 years of age with eosinophilic esophagitis. Press release. Sanofi. July 14, 2022. Accessed September 27, 2023. https://www.sanofi.com/en/media-room/press-releases/2022/2022-07-14-05-00-00-2479427

3. Dupixent (dupilumab). Prescribing information. Regeneron; 2022. Accessed September 28, 2023. https://www.regeneron.com/downloads/dupixent_fpi.pdf