Despite impacting approximately 1% of the global population, there are no approved pharmacotherapies for celiac disease, and disease management relies on patients’ adherence to a strict gluten-free diet.

Persistent or recurrent malabsorptive symptoms are observed in many patients, despite strict adherence to this diet, which underlines the unmet need for novel drug therapies.

The rise of gluten sensitivity

The prevalence of celiac disease has risen rapidly in recent decades, which has largely been attributed to changes in environmental factors including changing patterns of infection, as well as changing patterns of gluten consumption.

This has boosted interest and investment in celiac disease drug development, as companies attempt to capitalise on an increasingly large untapped market.

There are currently 22 pipeline products in active development for celiac disease. Of these, nine products have reached clinical development including four Phase I products and five Phase II products. A range of molecules types is represented among the pipeline products, the majority of which are biologics. These include proteins, peptides, monoclonal antibodies, and vaccines.

Celiac disease pipeline by stage of development and molecule type

Source: GlobalData
Note: mAb = monoclonal antibody

Although relatively modest in size, the pipeline is innovative and diverse in terms of the molecular targets and mechanisms of action represented. Of the 22 products in development, 13 have a disclosed molecular target, all of which are first-in-class. This is a highly encouraging trend in terms of the potential for these investigational drugs to progress to breakthrough therapies.

In the pipeline, late-stage drugs

Among the most promising late-stage drugs is IB Pharmaceutical’s larazotide acetate, a tight junction regulator currently in Phase II development.

By antagonising proteinase-activated receptor 2 and epidermal growth factor receptor, the drug appears to promote tight junction assembly and actin filament re-arrangement, which prevents gluten from reaching the intestinal submucosa and triggering an inflammatory response.

A Phase IIb study that investigated larazotide acetate as an adjunct to a gluten-free diet was the first large therapeutic trial in celiac disease to meet its primary endpoint of reducing signs and symptoms of the disease.

According to GlobalData’s drug sales database, larazotide acetate is expected to launch in 2021 and reach $217 million in sales by 2024.

Forthcoming report:
GlobalData (2019). Frontier Pharma: First-in-Class Innovation in Gastrointestinal Disorders, to be published.