The eosinophilic esophagitis (EoE) therapeutics market has considerable unmet needs; notably, there will be limited clinical developments for pediatric patients in the future. Last year, the US had 1,170,618 lifetime diagnosed prevalent cases of EoE, including 139,440 paediatric cases (aged 11 years and younger) and 1,031,178 adolescent and adult cases (aged 12 years and older).
EoE occurs in children and adults, with symptoms often non-specific and dependent on the age of onset. EoE symptoms in children include difficulty feeding, difficulty eating, vomiting, abdominal pain, decrease in appetite, difficulty swallowing (dysphagia), food impaction and a failure to thrive. In adolescents and adults, EoE symptoms appear as dysphagia, food impaction, chest pain that is often centrally located and does not respond to antacids, and backflow of undigested food (regurgitation).
At present, paediatric patients receive off-label proton pump inhibitors, steroids and dietary therapy similar to adolescents and adults. Unlike adolescents and adults, however, late-stage pipeline agents for EoE are not being studied in paediatric populations, which suggests that they will not be eligible to receive them. Figure 1 illustrates the six EoE pipeline agents currently in Phase III development in the US.
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 aged 1–11 years. Although Dupixent is expected to be the first marketed drug for paediatrics, the drug is anticipated to target a small subgroup of patients who do not respond to other therapies, rather than being a first-line therapy.
Key opinion leaders interviewed by GlobalData strongly encouraged the initiation of clinical trials for paediatric EoE patients and suggested easing the inclusion and exclusion criteria to improve enrolling paediatric cases in clinical trials. In addition, last September, the US Food and Drug Administration (FDA) finalised 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 paediatric-specific considerations. The guidance also recommended that the adolescent trials should be followed by trials for paediatric patients aged younger than 12 years, guided by safety and efficacy data from adolescent and adult trials.
GlobalData expects that limited treatment options for paediatric patients will remain a significant unmet need in the EoE space. As a result, the opportunity remains for developers to expand the current clinical studies to paediatric populations.