Current Status of Celiac Disease Drug Development

Am J Gastroenterol. 2016 Jun;111(6):779-86. doi: 10.1038/ajg.2016.105. Epub 2016 Mar 29.

Abstract

Celiac disease (CeD) is one of the most common immune-mediated diseases. Symptoms and disease activity are incompletely controlled by the gluten-free diet, which is currently the only available therapy. Although no therapies are yet approved, there is a growing field of candidates and an improving understanding of the regulatory pathway. In this review, we briefly discuss the epidemiology, pathophysiology, and current treatment paradigm for CeD. We also review the major classes of therapies being considered for CeD and discuss extensively what is known and can be surmised regarding the regulatory pathway for approval of a CeD therapeutic. The coming years will see an increasing number and diversity of potential therapies entering clinical trials and hopefully the first approved agents targeting this significant unmet medical need. Although biomarkers including histology and serology will always be important in therapeutic clinical trials, they currently lack the necessary evidence linking them to improved patient outcomes required for use as primary outcomes for drug approval. For this reason, patient-reported outcomes will likely be primary end points in Phase III CeD trials for the foreseeable future.

Publication types

  • Review

MeSH terms

  • Biomarkers
  • Celiac Disease / drug therapy*
  • Celiac Disease / epidemiology
  • Celiac Disease / physiopathology
  • Combined Modality Therapy
  • Diet, Gluten-Free
  • Drug Discovery*
  • Gastrointestinal Agents / therapeutic use*
  • Humans

Substances

  • Biomarkers
  • Gastrointestinal Agents