A phase 3 trial of dupilumab (Dupixent; Sanofi and Regeneron) has shown positive results for use in children aged 1 to 11 years afflicted with eosinophilic esophagitis.
Promising results have arisen from a phase 3 trial investigating dupilumab (Dupixent; Sanofi and Regeneron) for eosinophilic esophagitis (EOE) in children aged 1 to 11 years.
Along with EOE, dupilumab is used to treat patients with atopic dermatitis, asthma, andchronic rhinosinusitis with nasal polyposis. The US Food and Drug Administration approved the use of dupilumab in patients with EoE aged 12 years and older in May 2022.
EOE is a chronic inflammatory disease that causes damage to the esophagus, preventing it from working properly. Results from prior studies have shown that interluekin (IL-)4 and IL-13 are key factors in the type 2 inflammation leading to EOE. Symptoms include acid reflux, vomiting, abdominal discomfort, trouble swallowing, and a failure to thrive.
Children with EOE will experience roadblocks in development due to these symptoms, and potentially develop an anxiety toward food that remains as they grow older. Changes to one’s diet are the most common form of treatment, but dupilumab is used to alleviate symptoms as well.
The phase 3 trialfor dupilumab consisted of 102 children aged 1 to 11 years, who were assigned to receive either a higher or lower dose based on their body weight, or a placebo. Significant histological disease remission was achieved in 68% of children on the higher dose and 58% of children on the lower dose on week 16, while 3% of children on the placebo achieved this result.
Peak esophageal intraepithelial eosinophil count from baseline saw an 86% decrease in children who received dupilumab, while those who received placebo experienced a 21% increase. Reductions in abnormal endoscopic findings and disease severity and extent were also seen in the children on dupilumab.
Caregivers recorded an improvement in the proportion of days where children experienced symptoms from baseline, and body weight for age percentile increased by about 3%. Investigators also saw improvements for secondary endpoints similar to those from the primary endpoints. These results were consistent with those from prior studies on patients aged 12 years and above.
“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,” said George D. Yancopoulos, MD. “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.”
Adverse events in dupilumab compared to the placebo included mild to moderate COVID-19, rash, headache, viral gastroenteritis, diarrhea,and nausea. Overall, adverse events were more often seen in the placebo group, and led to a 6% rate of treatment discontinuation.
Press Release: Dupixent (dupilumab) phase 3 trial shows positive results in children 1 to 11 years of age with eosinophilic esophagitis. Sanofi. July 14, 2022. Accessed August 19, 2022. https://www.sanofi.com/en/media-room/press-releases/2022/2022-07-14-05-00-00-2479427