Eosinophilic Esophagitis: Developing Drugs for Treatment Guidance for Industry | FDA

Eosinophilic Esophagitis: Developing Drugs for Treatment Guidance for Industry | FDA

FDA finalizes guidance, “Eosinophilic Esophagitis:  Developing Drugs for Treatment”

The U.S. Food and Drug Administration is announcing the finalization of a guidance for industry titled, “Eosinophilic Esophagitis: Developing Drugs for Treatment.” Eosinophilic Esophagitis (EoE) is a chronic immune or antigen-mediated disease characterized by white blood cell (eosinophil-predominant) inflammation of the esophagus. Left untreated, EoE leads to difficulty swallowing food, esophageal narrowing, and food getting stuck in the esophagus. Although there has been an increase in the number of development programs for the treatment of eosinophilic esophagitis (EoE), there are currently no FDA approved therapies for the treatment of EoE. The final guidance promotes successful development of drugs and therapeutic biologics for the treatment of patients with EoE by providing clarity on FDA expectations and recommendations. Specifically, the guidance provides the FDA’s current thinking regarding clinical trials and development programs for EoE drugs including recommendations for the necessary attributes of patients for enrollment, trial designs, efficacy considerations, safety assessments and pediatric considerations.

The final guidance details a change in our recommendations regarding how the Clinical Outcomes Assessment portion of the recommended co-primary endpoint data should be analyzed. This change is being made to promote interpretability both within individual development programs and across programs utilizing differing instruments as well as to increase power of the analyses. The final guidance also responds to  comments from the academic community and patient advocacy groups for FDA to provide clarity on other recommendations for trial design and will lead to resource savings to industry, the academic community, and the FDA by facilitating protocol development.

This guidance finalizes the draft guidance of the same name issued February 6, 2019. This guidance does not address the clinical development of drugs for the treatment of non-EoE, eosinophilic gastrointestinal disorders.