The European Medicines Agency EMA has approved dupilumab as a treatment foreosinophil esophagitis in children from 1 year of age. The approval specifically concerns children aged between 1 and 11 years weighing at least 15 kg and who are not adequately supervised, are not candidates for or are intolerant to conventional medical therapy. The decision extends the initial EU approval of dupilumab’s indication for EoE in adult and adolescent patients and makes dupilumab the first and only drug ever to be indicated for the treatment of these young patients. Dupilumab is also approved for this age group in the USA and Canada.
“Children affected by eosinophilic esophagitis who experience the disease in the first years of life are put to the test in their ability to eat,” declares Roberta Giodice, president of ESEO Italia. “The parents of these children have often relied on restrictive diets which do not adequately treat the disease and can hinder their growth at a critical time in their development which could impact them for years to come. We are happy that the research continues and offers new therapeutic options to improve the quality of their care.”
“Up to 50% of children with eosinophilic esophagitis in the European Union remain uncontrolled despite existing standard treatment options and, as a result, many of these young patients struggle to reach a weight appropriate for their growth stage due to severe symptoms such as difficulty swallowing and vomiting,” says Houman Ashrafian, MD, PhD, executive vice presidenthead of Research and Development at Sanofi. “This milestone provides an important new treatment for pediatric patients who, until now, have not had options specifically approved for their disease. Thanks to this new approach that specifically addresses the primary cause of eosinophilic esophagitis, dupilumab has the potential to offer these children a better prospect of improvement in their growth.”
The approval is based on the phase 3 study EoE Kids (Part A e Part B), conducted in children aged 1 to 11 years, which demonstrated that the response to dupilumab in children with EoE is similar to that obtained in approved adult and adolescent populations. In Part A, after 16 weeks, children who were treated with a higher dose of dupilumab in relation to their body weight had the following results compared to those treated with placebo:
- The majority achieved histological remission of the disease,endpoint primary. These results were maintained for up to one year in Part B of the study;
- There was a more than fourfold reduction in peak esophageal intraepithelial eosinophil count from baseline;
- There was a reduction in abnormal endoscopic findings and in the severity and extent of disease (measured at the microscopic level).
- There was an improvement in the frequency and severity of EoE symptoms and a (numerical) reduction in days with at least 1 symptom, based onoutcome reported by caregiver.
The most common side effects in all indications include injection site reactions, conjunctivitis, allergic conjunctivitis, arthralgia, herpes oral and eosinophilia. In patients aged 1 to 11 years, the most commonly observed side effects with dupilumab in both weight-based dosing regimens compared to placebo during Part A were COVID-19, nausea, injection site pain, and headache.
“Eosinophilic esophagitis presents a unique challenge for young children, who struggle with the ability to feed themselves at a time in life when proper nutrition is essential to their growth and development,” says George D. Yancopoulos, MD, Ph .D., Board co-chairpresident and chief scientific officer of Regeneron. “This approval confirms the efficacy and safety profile of dupilumab, already established in patients with larger EoE.”
EOSINOPHIL ESOPHAGITIS
EoE is a chronic, progressive inflammatory disease associated with type 2 inflammation that damages the esophagus and prevents it from functioning properly. Diagnosis is often difficult, as it is confused with other more common digestive conditions with similar symptoms, causing delays in diagnosis. EoE can severely affect a child’s ability to eat and can also cause vomiting, abdominal pain, difficulty swallowing, decreased appetite, and growth problems. Ongoing management of EoE may be necessary to reduce the risk of complications and disease progression.
