In April, the U.S. Food and Drug Administration (FDA) released the long-awaited draft Guidance, “Celiac Disease: Developing Drugs for Adjunctive Treatment to a Gluten-Free Diet.” This draft Guidance provides recommendations for sponsors for the clinical development of drugs for adults for the treatment of celiac disease as an adjunct to the gluten-free diet. An adjunct to the gluten-free diet is a treatment that would address accidental gluten exposure, such as in cross-contamination with food that is not gluten-free, by decreasing intestinal inflammation and symptoms. 

The draft Guidance does not address the development of celiac disease drugs to prevent symptoms only or to allow patients to freely consume gluten (known as a “monotherapy”). It also does not address the development of celiac disease drugs for patients who are asymptomatic or with minimal histologic inflammation (intestinal damage) and who continue to experience symptoms. 

The draft Guidance does state what endpoints, or therapeutic outcomes, FDA will be assessing when evaluating the effectiveness and safety profile of any proposed therapy that is meant to supplement the gluten-free diet, and how they want those endpoints measured.  

The release of the draft Guidance was greeted by a diverse and passionate range of opinions within the celiac disease research community. Immediately, the Celiac Disease Foundation was contacted by a number of key stakeholders, and, in response, offered to host a meeting at Digestive Disease Week during the Society for the Study of Celiac Disease (SSCD) annual meeting to foster an honest and open exchange of ideas with biopharmaceutical companies currently in celiac disease drug development and clinician key opinion leaders. Celiac Disease Foundation Board Member, Kristin Yarema, PhD, Chief Commercial Officer of Atara Biotherapeutics and parent of a child with celiac disease, and Dan Adelman, MD, Celiac Disease Foundation Research Committee Chair, provided testimony. 

At the well-attended meeting in San Diego, with stakeholder representatives from across the country and internationally on livestream, it was decided that the SSCD would form a working group to draft for FDA a consensus statement from the celiac disease research community, with Marilyn G. Geller, Celiac Disease Foundation CEO, providing comments on behalf of the patient community.  

Celiac disease is the most prevalent autoimmune disorder to still lack an FDA-approved treatment. With gluten in more than 80% of our foodstuffs, and up to 50% of patients reporting ongoing symptoms despite their best efforts to follow a strict gluten-free diet, patients are demanding adjunct treatments. Some are concerned that the draft Guidance will limit the pool of celiac disease patients eligible to enroll in a clinical trial. The Celiac Disease Foundation appreciates that the criteria outlined for enrolling patients is rigorous in order to ensure the unequivocal safety and efficacy of future treatments, and that treatments must be tested in a population that has objectively measurable celiac disease. The Foundation also understands that in a perfect world devoid of gluten exposure, a therapeutic treatment would not be necessary, but that in the real world, where very few can achieve a 100% gluten-free diet, adjunct therapeutic treatments are imperative. 

The SSCD Working Group was tasked with determining what clarification to the draft Guidance is needed to ensure the speedy development of drugs for adjunctive treatment to a gluten-free diet, making certain that FDA understands and appreciates what the patient community wants and needs while not sacrificing safety and efficacy. The Celiac Disease Foundation submitted additional comments to ensure that the final Guidance allows for sufficient patients to enroll in clinical trials for celiac disease treatments, so that all with celiac disease may have access should they desire an adjunct treatment to their gluten-free diet. The public is invited by FDA to comment as well. All comments are due by June 14. 

A copy of the Guidance is linked here for your reading. You may review the Celiac Disease Foundation and SSCD draft Guidance comments here.

As always, if you have questions or comments about the draft Guidance, please reach out to us at [email protected]. It is important that we hear from you. We look forward to updating you once FDA has provided the final Guidance.