Limited treatment options for pediatric patients in the US remain a significant unmet need in the eosinophilic esophagitis (EoE) space, providing an opportunity for developers to expand the current clinical studies to pediatric populations, finds GlobalData, a leading data and analytics company.

In 2020, the US had 1.2 million lifetime diagnosed prevalent cases of EoE, including 139,440 pediatric cases (ages 11 and younger) and 1 million adolescent and adult cases (ages 12 and older). EoE occurs in children and adults, and symptoms are often non-specific and depend on the age of onset.

Mandana Emamzadeh, PhD, Pharma Analyst at GlobalData, comments: “Currently, pediatric patients receive off-label proton pump inhibitors, steroids, and dietary therapy similar to adolescents and adults. However, unlike adolescents and adults, the late-stage pipeline agents are not being studied in pediatric populations, which suggests that they will not be eligible to receive them.”

Of the six pipeline agents in late-stage development, Sanofi and Regeneron Pharmaceuticals’ Dupixent (dupilumab) is the only pipeline agent being studied in Phase III for a younger population (ages 1–11 years old). Although Dupixent is expected to be the first marketed drug for pediatrics, the drug is anticipated to target a small subgroup of patients who do not respond to other therapies, rather than as a first-line therapy.

Emamzadeh continues: “Key opinion leaders (KOLs) interviewed by GlobalData strongly encouraged the initiation of clinical trials for pediatric EoE patients and suggested easing the inclusion and exclusion criteria to improve enrolling pediatric cases in clinical trials.”

Additionally, in September 2020, the FDA finalized guidance to assist sponsors in the clinical development of drugs for the treatment of EoE. Specifically, this guidance addresses the FDA’s current thinking regarding clinical trials and development programs for EoE drugs, including recommendations for the necessary attributes of patients for enrollment, trial designs, efficacy considerations, safety assessments, and pediatric-specific considerations. The guidance also recommended that the adolescent trials should be followed by trials for pediatric patients younger than 12 years, guided by safety and efficacy data from adolescent and adult trials.