Fasenra Granted Orphan Drug Designation

AstraZeneca announced the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Fasenra (benralizumab) for the treatment of eosinophilic oesophagitis (EoE).

EoE is a rare, chronic, inflammatory disease that occurs when eosinophils, a type of white blood cell, accumulate in the oesophagus causing injury and inflammation. 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.

"Eosinophilic oesophagitis is an allergic inflammatory disease that can affect children and adults, and typically patients experience a range of debilitating symptoms including severe pain and difficulty swallowing food," Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, said. "Currently there are no FDA-approved treatments for eosinophilic oesophagitis. Because Fasenra depletes eosinophils in blood and tissue, it could become a potential new medicine to treat patients with this rare disease."

Fasenra is AstraZeneca's first respiratory biologic medicine and is currently approved as an add-on maintenance treatment for severe, eosinophilic asthma in the US, EU, Japan and other countries. In November 2018, the FDA granted ODD for Fasenra for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA), and also granted ODD for the treatment of hypereosinophilic syndrome (HES) in February 2019.

EoE is a rare, chronic, inflammatory disease of the oesophagus characterized by the accumulation of eosinophils, a type of white blood cell that are a normal part of the immune system, in the oesophageal lining tissue. The disease results in injury, fibrosis and dysfunction that if not effectively treated can make eating difficult or uncomfortable, potentially leading to chronic pain, difficulty swallowing, poor growth, malnutrition and weight loss.

The most common symptoms of EoE include reflux that does not respond to medication, difficulty swallowing, food becoming stuck in the oesophagus, nausea and vomiting, abdominal or chest pain, poor appetite and difficulty sleeping.

Patients are often treated with corticosteroids to manage inflammation. Currently there are no FDA-approved treatments for EoE.

Fasenra (benralizumab) is a monoclonal antibody that binds directly to the IL-5 receptor alpha on eosinophils and attracts natural killer cells to induce rapid and near-complete depletion of eosinophils via apoptosis (programmed cell death).

Fasenra is AstraZeneca’s first respiratory biologic, now approved as an add-on maintenance treatment in severe eosinophilic asthma in the US, EU, Japan and other countries, with further regulatory reviews ongoing. In June 2019, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for Fasenra self-administration and the Fasenra pen, a new auto-injector delivery device. Fasenra is in development for severe nasal polyposis, eosinophilic oesophagitis, eosinophilic granulomatosis with polyangiitis, hypereosinophilic syndrome and chronic obstructive pulmonary disease.

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