The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Fasenra (benralizumab) for the treatment of hypereosinophilic syndrome (HES).
HES is a group of rare, potentially fatal disorders characterized by high numbers of eosinophils in blood and tissues, which can cause progressive damage to any organ in the body. The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.
A Phase II clinical trial of Fasenra for the treatment of HES has been conducted by the United States National Institutes of Health in collaboration with AstraZeneca, with results expected to be published later in 2019. In the trial, Fasenra depleted blood eosinophils at week 12 compared with placebo, the primary endpoint of the trial, with evidence of eosinophil clearance in affected tissue at week 24.
“In patients with hypereosinophilic syndrome, high levels of eosinophils contribute to a range of debilitating symptoms and can even lead to life-threatening organ damage,” Mene Pangalos, Executive Vice President, R&D BioPharmaceuticals, said. “Based on results from the Phase II trial, we believe Fasenra has the potential to address critical unmet medical needs in patients living with hypereosinophilic syndrome.”
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Fasenra is AstraZeneca’s first respiratory biologic and is currently approved as an add-on maintenance treatment for severe, eosinophilic asthma in the US, EU, Japan and several other countries. In November 2018, the FDA granted ODD for Fasenra for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA).
HES is a group of rare disorders in which high numbers of eosinophils are found in the blood and tissue which can cause progressive organ damage over time, and if left untreated, can be fatal. HES most commonly impacts the skin, heart, lungs, gastrointestinal tract and central nervous system.
The symptoms of HES may include cough, fever, fatigue, asthma, difficulty breathing, wheezing, recurrent upper respiratory tract infections, abdominal pain, vomiting, diarrhea, skin rashes, arthritis, muscle aches and joint pain.
The goal of HES treatment is to reduce eosinophils in the blood and tissues, prevent organ damage and slow disease progression. HES treatment typically includes glucocorticoids, immunomodulatory therapies and cytotoxic therapies.
Fasenra (benralizumab) is a monoclonal antibody that binds directly to IL-5 receptor α 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 several other jurisdictions, with further regulatory reviews ongoing. Where approved, Fasenra is available as a fixed-dose subcutaneous injection via a prefilled syringe administered once every 4 weeks for the first 3 doses, and then once every 8 weeks thereafter. Fasenra is also being studied in severe nasal polyposis. Phase III trials for Fasenra in HES have not yet commenced.
Fasenra was developed by AstraZeneca with MedImmune, the company’s global biologics research and development arm, and is in-licensed from BioWa, a wholly-owned subsidiary of Kyowa Hakko Kirin, Japan.