Marc Rothenberg, MD, PhD: EoE Receives First FDA Approval with Dupilumab

Marc Rothenberg, MD, PhD

Last week the US Food and Drug Administration (FDA) approved dupilumab (Dupixent) for the treatment of eosinophilic esophagitis (EoE) in adult and pediatric patients 12 years and older, weighing at least 40 kg.

This approval marks the first treatment indicated for EoE in the US. The rare condition affects people of all ages and genders with a higher incidence among males. As of right now, dupilumab is the sole biologic to achieve positive, clinically meaningful phase 3 results in this patient population.

"This is a victory for science, patients, rare diseases and advocacy," Marc Rothenberg, MD, PhD, director of the Cincinnati Center for Eosinophilic Diseases at Cincinnati Children’s, said in a statement.

Rothenberg has been studying EoE for the past 2 decades and his research has played an essential role in the development of dupilumab for EoE treatment. He and his team were able to demonstrate that EoE was mediated by an allergic response to food, and the disease mechanism involved a dysregulated immune response driven by interleukin (IL-13) elicited inflammation.

As dupilumab achieved FDA approval, Rothenberg offered to answer some questions about the treatment for HCPLive.

Dupilumab is the first treatment in the US to be approved for patients 12 years and older with EoE. How will this approval shift treatment practices for this condition?

Rothenberg: At present, patients are treated with restricted elimination diets, as well as off-label usage of proton pump inhibitors (PPIs), as well as topical glucocorticoids (asthma drugs made for inhalation) but now swallowed. These drugs do not work in everyone, patients universally relapse upon withdrawal of medicines, and diet sensitization changes over time. 

As such, there is a need for approved and better therapy. Dupilumab will be that answer for many. It improves symptoms, pathology, endoscopy, and molecular changes in the esophagus. It will also be a relief to know that the FDA has finally approved a drug for EoE.

Will most patients adopt this as part of their treatment regimen if they haven’t already?

Rothenberg: It will likely be used in a subset of patients who are unsatisfied with their current therapy. Also, not everyone, with or without insurance, will be able to afford it.

In what ways has dupilumab succeeded where competitors have not?

Rothenberg: It is the first drug to improve symptoms plus histology (decrease eosinophil counts in the esophagus).

Do you think there’s motivation to get this treatment approved for patients under the age of 12?

Rothenberg: Yes, there is strong motivation, and a large-scale study is currently underway and almost complete.

Rothenberg continues to lead studies of EoE and related conditions as principal investigator of the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR), which is part of the NIH-funded Rare Diseases Clinical Research Network (RDCRN).