First and only investigational Phase 3 trial to show positive results in these young children; results follow recent approval of Dupixent in people with eosinophilic esophagitis aged 12 years and older who weigh at least 40 kilograms
Trial met its primary endpoint, with 68% of patients on higher dose Dupixent and 58% on lower dose Dupixent achieving histological disease remission at 16 weeks
Fifth pediatric pivotal trial across three type 2 inflammatory diseases to reinforce the well-established efficacy and safety profile of Dupixent
TARRYTOWN, N.Y. and PARIS, July 14, 2022 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that a Phase 3 trial assessing the investigational use of Dupixent® (dupilumab) in children aged 1 to 11 years with eosinophilic esophagitis (EoE) met its primary endpoint of histological disease remission at 16 weeks with both higher and lower dose weight-tiered regimens. There are no approved treatments for children with EoE under 12 years of age.
"Dupixent is the first medicine to alleviate key signs of eosinophilic esophagitis in children as young as 1 year of age in a Phase 3 trial. The efficacy of Dupixent demonstrates that in this age group, as in adults, IL-4 and IL-13 are key drivers of the type 2 inflammation underlying this debilitating disease," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron, and a principal inventor of Dupixent. "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. The positive results from this Phase 3 pediatric trial show Dupixent has the potential to improve signs of eosinophilic esophagitis and support healthy weight gain in children from their first birthday."
EoE is a chronic inflammatory disease that damages the esophagus and prevents it from working properly. The results seen with Dupixent in adults and children with EoE demonstrate that IL-4 and IL-13 are key drivers of the type 2 inflammation underlying this disease. In children, common symptoms of eosinophilic esophagitis include acid reflux, vomiting, abdominal discomfort, trouble swallowing, and a failure to thrive. These symptoms can impact growth and development, and can cause food-related fear and anxiety which can persist through adulthood. Diet adjustments, which oftentimes include the elimination of many foods, is the standard treatment for EoE, as well as the use of treatments not approved for the disease. These include proton pump inhibitors, swallowed topical corticosteroids, or in severe cases, a feeding tube, which may be used to ensure proper caloric intake and weight gain. Of the approximately 21,000 children under the age of 12 in the U.S. currently being treated for EoE, about 9,000 do not satisfactorily respond to the unapproved therapies they have been treated with and potentially require advanced therapy.
"We are incredibly excited to share results from this Phase 3 pivotal trial evaluating Dupixent in young children suffering from eosinophilic esophagitis – the first ever to show positive results across a variety of primary and secondary endpoints. The lack of treatment options for children living with eosinophilic esophagitis leaves many caregivers with the stress and burden of adapting their child's meals and their entire family's daily schedules to ensure healthy growth and development. In some cases, they must resort to off-label use of poorly studied treatments like steroids that can pose serious health risks when used long term," said Naimish Patel, M.D., Senior Vice President, Head of Global Development, Immunology and Inflammation at Sanofi. "The faster and larger than anticipated enrollment in this trial further emphasizes the unmet treatment needs for children with EoE and underscores the significance of these first-ever positive results."
In the Phase 3 trial, 102 children aged 1 to 11 were randomized to receive Dupixent, in either a higher dose (n=37) or lower dose (n=31) regimen based on body weight, or placebo (n=34). At 16 weeks, 68% of children on higher dose and 58% of patients on lower dose Dupixent achieved the primary endpoint of significant histological disease remission (peak esophageal intraepithelial eosinophil count of ≤6 eosinophils [eos]/high power field [hpf]) compared to 3% of children on placebo (p