The European Medicines Agency (EMA) has granted approval to Sanofi and Regeneron's Dupixent (dupilumab) for the treatment of eosinophilic esophagitis (EoE) in children aged one to 11 years, weighing a minimum of 15kg, who have not responded adequately to conventional medicinal therapies. This decision marks a significant advancement, providing the first and only approved medication for this young patient demographic within the European Union. The approval is based on data from the pivotal Phase III EoE KIDS trial, a randomized, double-blind, placebo-controlled study demonstrating Dupixent's efficacy and safety in pediatric EoE patients.
Clinical Trial Data
The EMA's approval is rooted in the outcomes of the EoE KIDS trial. Part A of the study involved 71 children who were administered a weight-based dosing regimen of Dupixent. After a 16-week treatment period, 68% of the patients in the Dupixent group achieved the primary endpoint of histological disease remission, compared to only 3% in the placebo group. These positive outcomes were sustained for up to one year in Part B of the study. Part C, an open-label extension phase spanning 108 weeks, was recently completed to evaluate longer-term outcomes.
Mechanism of Action
Dupixent, developed using Regeneron’s VelocImmune technology, is a fully human monoclonal antibody. It functions by specifically blocking the interleukin-4 (IL-4) and interleukin-13 (IL-13) signaling pathways, which are key drivers of the inflammation associated with EoE.
Impact on Patient Care
According to Sanofi's Executive Vice-President, Houman Ashrafian, up to half of all children in the EU with eosinophilic esophagitis remain uncontrolled despite existing standard of care treatment options. As a result, many of these young patients struggle to maintain weight due to serious symptoms such as vomiting and difficulty swallowing. This approval provides an important new treatment for pediatric patients who were previously without options specifically approved for their disease.
