FDA Grants Dupilumab Breakthrough Therapy Designation for Eosinophilic Esophagitis

The US Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation for dupilumab (Dupixent) to treat eosinophilic esophagitis (EoE).

The designation will expedite the development and review of the drug developed by Regeneron Pharmaceuticals and Sanofi for the treatment of patients at least 12 years old.

The designation is based on the positive result from part A of a phase 3 trial.

Currently, there are no FDA-approved treatments for eosinophilic esophagitis.

In the randomized, double-blind, placebo-controlled trial, investigators examined 81 patients with EoE. The researchers met both of their co-primary endpoints, as well as all of the key secondary endpoints.

Patients in the study were treated weekly with dupilumab 300 mg over a 24-week period. The patients experienced a reduction in symptoms, esophageal inflammation, and abnormal endoscopic findings in the esophagus.

The trial also demonstrated safety results consistent with the known safety profile of the study drug in other approved indications, such as atopic dermatitis, maintenance therapy for moderate-to-severe eosinophilic or oral steroid dependent asthma, and chronic rhinosinusitis with nasal polyposis.

EoE is a chronic and progressive type 2 inflammatory disease that damages the esophagus, preventing the organ from working properly. Over time excessive type 2 inflammation can cause scarring and narrowing of the esophagus, making it challenging for patients to swallow.

When untreated, the disease can impact a patient’s ability to eat, causing food to become stuck after being swallowed. There are currently about 160,000 patients with eosinophilic esophagitis in the US being treated with various unapproved therapies or diet modification. Of this patient population, approximately 50,000 individuals have failed multiple treatments.

In 2017, Dupixent also was granted Orphan Drug Designation for the potential treatment of EoE.