Rare Disease Report

Breakthrough Therapy Designation Given to Eosinophilic Esophagitis Drug

JUNE 14, 2016
James Radke, PhD
Eosinophilic esophagitis characterized by food getting stuck in patient’s throat.

The FDA has granted Breakthrough Therapy Designation to Shire’s SHP621 (budesonide oral suspension) for eosinophilic esophagitis (EoE).
EoE is a chronic disease with an estimated prevalence in the United States of approximately181,000—putting it just under the 200,000 cut off point for a rare disease. EoE is characterized by inflammation and accumulation of eosinophils in the esophagus. Patients with eosinophilic esophagitis may have persistent or relapsing symptoms related to esophageal dysfunction, which include dysphagia (difficulty swallowing) and food impaction.
The dysphagia is usually associated with solid food sticking to the esophagus. Often the only remedy is for the person to regurgitate the food if it does not pass through to the stomach. On rare occasions, the food can be impacted. At which point, endoscopy will be necessary to remove the food.

At present, there are no orphan approved drugs for eosinophilic esophagitis  but recently, Receptos received orphan drug designation for their monoclonal antibody RPC4046 that is in phase 2 studies.

What is Breakthrough Therapy Designation?

 Breakthrough Therapy Designation is granted to a therapy that is intended to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over current standard of care. Under the designation, the FDA provides intensive guidance, organizational commitment involving senior managers, and eligibility for rolling and priority review of the application.  

About Budesonide Oral Suspension

budesonide oral suspension is formulated specifically for EoE. In a Phase 2 placebo-controlled trial involving adolescents and adults with EoE, the study found that 12 weeks of budesonide oral suspension treatment significantly reduced dysphagia symptoms and increased the proportion of patients with a histologic response. It was based on this Phase 2 trial that the FDA granted Breakthrough Therapy Designation. 
A Phase 3 trial is currently underway. For more information about the trial, click here or here.

Image courtesy wikimedia commons.

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