AstraZeneca biologic gets orphan drug status for rare blood disease


TheUS Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) forAstraZeneca’s Fasenrafor 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 the 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.

Sean Bohen, executive vice president, Global Medicines Development and chief medical officer, said: “EGPA is a rare, but debilitating inflammatory disease and patients with the disease typically have very high levels of eosinophils. Our clinical trials forFasenrain 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.”

EGPA is characterized by inflammation of blood vessels and the presence of elevated levels of eosinophils, a type of white blood cell.Fasenrainduces rapid and near-complete depletion of eosinophils in the blood and has shown promise in treating severe, eosinophilic asthma.

Fasenrais 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 other jurisdictions.

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 from medicine.

Fasenrais 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).

Where approved,Fasenrais available as a fixed-dose subcutaneous. Phase III trials forFasenrain EGPA have not commenced.

AstraZeneca employs 1,500 in Delaware.

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