FDA Grants AstraZeneca's Tezepelumab Orphan Drug Designation

Published on: 

AstraZeneca’s tezelpelumab was granted orphan drug designation by FDA for the treatment of eosinophilic esophagitis.

Astrazeneca announced on Oct. 8, 2021that FDA granted its drug, tezepelumab, orphan drug designation for the treatment of eosinophilic esophagitis (EoE). EoE is a rare, chronic, inflammatory disease that has no approved treatments to date.

EoE occurs when eosinophils, a type of white blood cell, accumulate in the esophagus. If left untreated, it can make eating difficult or uncomfortable, leading to chronic pain, difficulty swallowing, poor growth, malnutrition, and weight loss. Other cells, including mast cells, T-cells, and fibroblasts, may also drive injury, inflammation, and detrimental tissue remodeling.

According to a company press release, tezepelumab is a monoclonal antibody that inhibits the action of thymic stromal lymphopoietin (TSLP), a key epithelial cytokine that sits at the top of multiple inflammatory cascades. The intent behind blocking TSLP is to prevent pro-inflammatory cytokines by immune cells, which are implicated in many epithelial disease pathophysiologies.

“Eosinophilic esophagitis is a rare disease which involves a range of inflammatory cells that contribute to debilitating symptoms for patients, including severe pain and difficulty swallowing food,” said Mene Pangalos, executive vice-president, BioPharmaceuticals R&D, AstraZeneca, in the press release. “We are hopeful that tezepelumab, with its unique mechanism of action that targets the top of the inflammatory cascade, could become a potential new medicine to improve outcomes for these patients.”


AstraZeneca entered into a collaboration agreement with Amgen to develop tezepelumab in 2020. According to the press release, AstraZeneca leads development, while Amgen oversees the manufacturing process.

Source: AstraZeneca