FDA Grants Orphan Drug Designation to First-in-Class Treatment for Eosinophilic Esophagitis

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The immune-resetting peptide, developed by Revolo Biotherapeutics, received positive results from its Phase IIb study.

Revolo Biotherapeutics announced on Jan. 30, 2024 that FDA has granted Orphan Drug Designation to ‘1104, a first-in-class immune-resetting peptide being developed as a potential treatment for eosinophilic esophagitis (EoE). Revolo focuses on developing therapies that reset the immune system to achieve long-term remission for patients with autoimmune and allergic diseases. The original request for ‘1104 was submitted before its Phase IIa EoE study, and the request was amended following the study’s positive results.

The Orphan Drug Designation is granted by FDA to support the development and evaluation of new therapies that are intended for the treatment, diagnosis, or prevention of rare diseases and disorders affecting less than 200,000 people in the United States. EoE is a chronic, inflammatory disease in which eosinophils, specialized cells in the immune system, accumulate in the lining of the esophagus. This causes inflammation, resulting in progressive tissue injury. Untreated, patients can have difficulty swallowing, chronic pain, malnutrition, and weight loss. According to a study published in the American Journal of Gastroenterology, EoE affected around 23,000 patients in 2018, 6000 more than in 2015.

“We are excited that the FDA has granted ODD for ‘1104 in EoE, as there remains a significant unmet need for new treatment innovations for people living with this disease,” said Woody Bryan, president and chief executive officer of Revolo Biotherapeutics, in a Revolo press release. “We look forward to initiating a Phase IIb study of ‘1104 in EoE later this year.”


Evan Dellon, M.D., MPH, gastroenterologist, professor of Medicine and adjunct professor of Epidemiology at the University of North Carolina Chapel Hill, and Principal Investigator for the Phase IIa clinical trial, added, “’1104 is a promising EoE therapeutic with a novel mechanism of action.Based on the proof-of-concept data from the Phase IIa study, I am eager to see this molecule move forward into the next stage of development.”

Source: Revolo Biotherapeutics, The American Journal of Gastroenterology