USFDA grants Orphan Drug Designation to AstraZeneca Tezepelumab
UK: AstraZeneca has announced that Tezepelumab has been granted Orphan Drug Designation (ODD) in the US by the Food and Drug Administration (FDA) for the treatment of eosinophilic esophagitis (EoE).
Tezepelumab is being developed by AstraZeneca in collaboration with Amgen and is under Priority Review for patients with asthma in the US. The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.
EoE is a rare, chronic, inflammatory disease that occurs when eosinophils, a type of white blood cell, accumulate in the esophagus. In addition to eosinophils, other cells including mast cells, T-cells and fibroblasts drive injury, inflammation and detrimental tissue remodelling. If the disease is not effectively treated it can make eating difficult or uncomfortable, potentially leading to chronic pain, difficulty swallowing, poor growth, malnutrition and weight loss.
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: "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. There are currently no approved treatments for eosinophilic esophagitis in the US. 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."
A decision on tezepelumab's Priority Review in patients with asthma in the US is expected in the first quarter of 2022. Tezepelumab is also under regulatory review for asthma in the EU and Japan.
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