AstraZeneca PLC said Monday the US Food and Drug Administration has granted Orphan Drug Designation for Fasenra to treat EGPA, a rare autoimmune disease that can damage multiple organs and tissues.
Eosinophilic Granulomatosis with Polyangiitis, or EGPA, can be fatal without treatment. The disease can damage a patient’s lungs, skin, heart, gastrointestinal tract and nerves, and common symptoms include extreme fatigue, weight loss and muscle and joint pain.
The regulatory agency’s Orphan Drug Designation program is aimed at helping to facilitate the development of drugs for rare diseases that affect fewer than 200,000 people in the US.
EGPA is characterised by inflammation of blood vessels and elevated levels of eosinophils, a type of white blood cell. The drugmaker said Fasenra, an injectable treatment, is its 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.
Sean Bohen, AstraZeneca’s 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."
There are few effective medicines for EGPA, AstraZeneca said in a statement. Patients are often treated with chronic high-dose oral corticosteroids and can experience recurrent relapses when trying to taper off the medication.
Phase III trials for Fasenra in EGPA have not started, the company said. AstraZeneca developed Fasenra with MedImmune, its global biologics research and development arm, and is in-licensed from BioWa Inc., a subsidiary of Kyowa Hakko Kirin Co. Ltd. In Japan.