The FDA approval, which expands Dupixent’s initial approval granted in May 2022, for the treatment of EoE in patients aged 12 years and older, makes Dupixent as the first and only medicine approved in the US indicated for children with EoE
Regeneron Pharmaceuticals headquarters. (Credit: Ajay Suresh@Wikipedia)
The US Food and Drug Administration (FDA) has expanded the indication for Dupixent (dupilumab) to treat pediatric patients aged one to 11 years, weighing at least 15kg, with eosinophilic esophagitis (EoE).
Dupixent is jointly developed by US biotechnology company Regeneron and French pharmaceutical firm Sanofi, under a global collaboration agreement.
The current FDA approval expands the drug’s initial approval, granted in May 2022, for the treatment of EoE in patients aged 12 years and older, weighing at least 40kg.
With the expanded approval, Dupixent is the first and only medicine approved in the US, specifically indicated to treat paediatric patients with EoE.
Regeneron board co-chair, president and chief scientific officer and Dupixent principal inventor George Yancopoulos said: “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.
“With this approval, Dupixent becomes the first and only treatment option for EoE patients aged one year and older, weighing at least 15kg.
“By targeting the underlying type 2 inflammation that contributes to this disease, Dupixent has the potential to transform the standard of care for these children as it has for adults and adolescents with EoE.”
The FDA evaluated Dupixent for the expanded indication under Priority Review.
The expanded FDA approval is based on data from the Phase 3 EoE KIDS trial Part A and Part B, evaluating the efficacy and safety of the drug in children with EoE, aged one to 11 years.
In the study, 66% of children who received a higher dose of Dupixent at tiered dosing regimens based on weight achieved histological disease remission at 16 weeks, the primary endpoint.
The treatment resulted in 53% histological remission in children in Parts A and B, and 53% histological remission in children who switched to Dupixent from placebo in Part B.
In addition, the treatment also a greater decrease in the signs of EoE based on the Pediatric EoE Sign/Symptom Questionnaire-caregiver version (PESQ-C) in children.
Dupixent in children aged one to 11 years weighing at least 15kg showed a similar safety profile to that of the drug in adult and pediatric patients aged 12 years and above.
The most common adverse events include injection site reactions, upper respiratory tract infections, arthralgia and herpes viral infections.
In addition to the currently approved indications, Regeneron and Sanofi are studying dupilumab in a broad range of diseases driven by type 2 inflammation or other allergic processes.
Sanofi immunology and inflammation global development head Naimish Patel said: “Young children with eosinophilic esophagitis have significant unmet medical needs; despite existing treatment options, 40% of these children in the US under the age of 12 continue to experience symptoms of this disease.
“Today’s approval underscores our commitment to bringing therapies to young patients with unmet needs and also brings hope to these patients who are at a critical age where struggling to eat and maintain weight directly impacts their overall nutritional intake and development.”