Today, the US Food and Drug Administration (FDA) approved dupilumab (Dupixent) to treat eosinophilic esophagitis (EoE) in patients 12 years and older weighing at least 40 kilograms (approximately 88 pounds). This is the first treatment to be FDA approved for this condition.
EoE is a chronic, allergic inflammatory disease due to the buildup of eosinophils in the lining of the esophagus. Signs and symptoms vary but EoE patients often experience difficulty feeding, poor weight gain, heartburn, regurgitation, and dysphagia.
The approval of dupilumab was largely based on results from a phase 3 trial that included two 24-week treatment periods that were conducted independently in separate groups of patients. In Part A and Part B, patients received either placebo or 300 mg of dupilumab every week. The primary endpoints were the proportion of patients who achieved peak esophageal intraepithelial eosinophil count of ≤6 eosinophils per high-power field (eos/hpf) at week 24, and the change in the patient-reported Dysphagia Symptom Questionnaire (DSQ) score from baseline to week 24. The DSQ is a questionnaire designed to measure difficulty swallowing associated with EoE, with total scores ranging from 0 to 84; higher DSQ scores indicate worse symptoms.
In Part A of the trial, 60% of the 42 patients who received dupilumab achieved the pre-determined level of reduced eosinophils in the esophagus compared to 5% of the 39 patients who received a placebo. Patients in Part A who received dupilumab experienced an average improvement of 22 points in their DSQ score compared to 10 points in patients who received placebo.
In Part B, 59% of the 80 patients who received dupilumab achieved the pre-determined level of reduced eosinophils in the esophagus compared to 6% of the 79 patients who received a placebo. Patients in Part B who received dupilumab experienced an average improvement of 24 points in their DSQ score compared to 14 points in patients who received placebo.
The most common treatment emergent adverse events were injection site reactions, upper respiratory tract infections, joint pain, and herpes viral infections.
To learn more about EoE and other rare gastrointestinal disorders, visit checkrare.com/diseases/gastrointestinal-diseases/
