The European Commission has recently approved Dupixent® (dupilumab) as the first and only treatment option for young children aged 1 to 11 years with eosinophilic oesophagitis (EoE), offering new hope to families affected by this rare and often debilitating condition.

Previously, we reported on its approval in China for children under 12 years, marking a significant step forward for EoE treatment globally.

This EU approval is supported by data from the two-part EoE KIDS Phase 3 trial (Parts A and B), which established a "bridging" response, showing that children aged 1 to 11 with EoE respond to Dupixent in ways similar to the already-approved adult and adolescent populations.

The results of this trial were published in the New England Journal of Medicine (NEJM).

About the Dupixent Paediatric Eosinophilic Oesophagitis Trial 

The EoE KIDS Phase 3 trial was a randomised, double-blind, placebo-controlled study aimed at assessing the efficacy and safety of Dupixent in children aged 1 to 11 years with eosinophilic oesophagitis (EoE). The study consisted of two parts:

Part A enrolled 71 children, assigning them to higher-exposure (HE) or lower-exposure (LE) subcutaneous dupilumab regimens (tiered according to body weight) or to a placebo group for an initial 16-week period.

Part B extended to 36 weeks of active treatment, where eligible children in the Dupixent group continued with the treatment, while those previously receiving placebo switched to HE or LE dupilumab.

Participants in this trial had all been treated with conventional therapies, such as proton pump inhibitors and swallowed topical corticosteroids without achieving adequate relief, underscoring the need for new, targeted treatment options like Dupixent.

The primary endpoint was histologic remission at 16 weeks, with secondary endpoints assessing endoscopic and histopathologic measures of disease severity and caregiver-reported signs and symptoms of EoE.

The recently completed 108-week open-label extension period (Part C) was conducted to assess Dupixent's longer-term outcomes.

Trial  Results

A significantly higher percentage of patients in the dupilumab groups achieved histologic remission compared to those in the placebo group. The therapeutic effects of ongoing dupilumab treatment appeared to be sustained up to week 52.

HE dupilumab showed significant improvements across all key measures, including histologic, endoscopic, and transcriptomic outcomes, when compared to placebo.

The incidence of adverse events was comparable between the dupilumab and placebo groups, with most events being mild to moderate in severity.

Download NEJM Plain Language Summary

What is Eosinophilic Oesophagitis in Young Children?

Eosinophilic oesophagitis (EoE) is a chronic inflammatory disease where eosinophils, a type of white blood cell, accumulate in the oesophagus, leading to inflammation and tissue damage. In young children, EoE presents unique challenges, as it can cause feeding difficulties, failure to thrive, pain, and disrupted growth. 

Is Eosinophil Count the Main Marker to Measure Results?

While reducing eosinophil counts is important in treating young children with EoE, the real value of any treatment lies in providing meaningful symptom relief.

As evidenced in recent studies like the MESSINA trial with benralizumab, addressing eosinophil levels alone may not fully alleviate the most challenging symptoms, such as difficulty swallowing and feeding issues, which deeply impact the quality of life. 

EoE KIDS Phase 3 trial's focus on both histologic remission and caregiver-reported symptom improvement offers a more comprehensive approach to evaluate results. 

The findings highlight the ongoing need for EoE therapies that prioritise both inflammation control and symptom management, especially in young patients for whom these symptoms can affect growth and development.

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Read the full publication

Learn more about EoE symptoms in children

Benralizumab's Efficacy in Reducing Eosinophil Count in EoE. Messina Phase 3 Trial

Read more about EoE research with Prof. Rothenberg

Access to Dupixent (Dupilumab) Treatment in the UK Explained