The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to dupilumab (Dupixent®; Regeneron and Sanofi) for the treatment of eosinophilic esophagitis (EoE) in patients aged 12 years and older.
The designation was based on results from Part A of a double-blind, placebo-controlled, pivotal phase 3 trial that evaluated the effect of dupilumab in 81 patients aged ≥12 years with EoE. Patients were randomized to receive either dupilumab 300mg weekly or placebo for 24 weeks. The co-primary end points were the proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eosinophils per high-power field, and the absolute change in Dysphagia Questionnaire (DSQ) score, at 24 weeks.
Findings showed that patients treated with dupilumab met both co-primary end points achieving a statistically significant reduction in their esophageal eosinophilic count and an improvement in DSQ score compared with placebo. Moreover, dupilumab was associated with a statistically significant reduction in abnormal endoscopic findings (secondary end point). The safety profile of dupilumab was found to be similar to that seen in previous studies for approved indications.
Investigators are currently enrolling additional patients in Part B of the trial to assess dosing regimens, as well as Part C, a 28-week extended treatment period.
FDA grants Dupixent® (dupilumab) Breakthrough Therapy designation for eosinophilic esophagitis. https://www.prnewswire.com/news-releases/fda-grants-dupixent-dupilumab-breakthrough-therapy-designation-for-eosinophilic-esophagitis-301129713.html. Accessed September 14, 2020.