FDA Approves Dupilumab for Eosinophilic Esophagitis in Children Aged 1 to 11 Years

The US Food and Drug Administration on Thursday approved dupilumab (Dupixent; Regeneron and Sanofi) as the first treatment indicated specifically for children with eosinophilic esophagitis (EoE) aged 1 year to 11 years weighing at least 33 lbs (15 kg), according to a news release from Sanofi.

Granted under the FDA’s priority review designation, the approval expands on the Agency’s initial approval for EoE in May 2022 for youth aged 12 years and older, at a weight of at least 40 kg.

The chronic, progressive disease is driven by type 2 inflammation that damages the esophagus, disrupting proper function. Among the symptoms a child may experience are vomiting, heartburn, abdominal pain, trouble swallowing, food refusal, and failure to thrive, which can impact proper growth and development.

“Young children are some of the most vulnerable patients with eosinophilic esophagitis, or EoE, as this debilitating and progressive disease threatens their basic ability to eat. Until today, these children had no approved treatment options specifically for EoE, leaving many with unapproved medicines that failed to target the root cause of their disease,” said George D Yancopoulos, MD, PhD, board co-chair, president and chief scientific officer at Regeneron, and a principal inventor of dupilumab.

More than 20 000 children under the age of 12 years who have EoE may be receiving off-label treatment for the disease, according to Sanofi and Regeneron.

The FDA’s decision came after the Agency’s review of data from the 2-part phase 3 EoE KIDS (Parts A [16 weeks] and B [36-week extended active treatment]) trial that evaluated safety and efficacy of dupilumab in children aged 1 to 11 years with EoE.

At 16 weeks, among the children that received higher dose dupilumab at tiered dosing regimens based on weight (n = 32), 66% achieved the study’s primary endpoint, histologic disease remission, compared to 3% of who reached remission with placebo (n = 29). At 16 weeks investigators also reported “a greater decrease in the proportion of days with [1] or more signs of EoE based on Pediatric EoE Sign/Symptom Questionnaire-caregiver version (PESQ-C) was observed in children treated with [dupilumab]” compared to placebo, Sanofi stated in the press release.

At 52 weeks, 17 of 32 children (53%) treated with dupilumab in Parts A and B of the study sustained histologic remission. Among children who were switched to dupilumab from placebo in part B of EoE KIDS, 8 of the 15 in the cohort (53%) also sustained disease remission at study week 52.

“Young children with [EoE] have significant unmet medical needs,” Naimish Patel, MD, Sanofi head of global development, immunology and inflammation said in the Sanofi statement. “Despite existing treatment options, 40% of these children in the US under the age of 12 continue to experience symptoms of this disease.”

The most common adverse events (≥2%) observed more frequently with dupilumab vs placebo were injection site reactions, arthralgia, upper respiratory tract infections, and herpes viral infections, Sanofi said. One case of helminth infection was reported in the dupilumab arm in Part B of the EoE KIDS trial.

“By targeting the underlying type 2 inflammation that contributes to this disease, [dupilumab] has the potential to transform the standard of care for these children as it has for adults and adolescents with EoE,” Yancopoulos said in the press release.

Source: Dupixent FDA approved as first and only treatment indicated for children aged 1 year and older with eosinophilic esophagitis (EoE). News release. Sanofi. January 25, 2024. Accessed January 26, 2024. https://www.sanofi.com/en/media-room/press-releases/2024/2024-01-25-19-30-00-2817342