FDA grants Fasenra Orphan Drug Designation

AstraZeneca the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Fasenra (benralizumab) for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA).

EGPA is a rare autoimmune disease that can cause damage to multiple organs and tissues. 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.

“EGPA is a rare, but debilitating inflammatory disease and patients with the disease typically have very high levels of eosinophils,” Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer, said. “Our clinical trials for Fasenra in severe, eosinophilic asthma show it depletes eosinophils and we are exploring the potential of this medicine to address unmet medical needs in other eosinophil-driven diseases.”

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EGPA is characterized by inflammation of blood vessels and the presence of elevated levels of eosinophils, a type of white blood cell. Fasenra induces rapid and near-complete depletion of eosinophils in the blood and has proven efficacy in severe, eosinophilic asthma, which suggests it may benefit patients with EGPA.

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 jurisdictions.

EGPA, formerly known as Churg-Strauss Syndrome, is a rare, chronic autoimmune disease that is caused by inflammation of small to medium-sized blood vessels. EGPA can result in damage to multiple organs, including lungs, skin, heart, gastrointestinal tract and nerves. The most common symptoms include extreme fatigue, weight loss, muscle and joint pain, rashes, nerve pain, sinus and nasal symptoms, and shortness of breath. Without treatment, the disease may be fatal.

Elevated levels of eosinophils play a central role in EGPA disease pathophysiology. All patients with EGPA have very high levels of eosinophils at some point in their disease, both in peripheral blood and in affected tissues or organs. People with EGPA usually have asthma that may have developed as an adult, and often have sinus and nasal symptoms.

There are few effective medicines for EGPA. Patients are often treated with chronic high-dose oral corticosteroids (OCS) and can experience recurrent relapses when attempting to taper off OCS.

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