Issue: March 2020

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February 11, 2020
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FDA Grants Orphan Drug Status to IV Immunoglobulin Treatment for Systemic Sclerosis

Issue: March 2020
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The FDA has granted orphan drug designation to an investigational intravenous immunoglobulin therapy for the treatment of systemic sclerosis, according to a press release from CSL Behring.

Currently approved to treat patients with primary immunodeficiency and chronic immune thrombocytopenic purpura, the IV immunoglobulin treatment (Privigen) received fast track designation in October 2019 to treat serious pulmonary, skin and musculoskeletal manifestations of systemic sclerosis.

“CSL Behring is driven by our promise to develop and deliver innovative therapies for patients with the highest unmet need,” Mittie Doyle, vice president of Research and Development, Immunology and Neurology Therapeutic Area at CSL Behring, said in a press release. “Receiving orphan drug designation for Privigen as an investigational SSc therapy is an important milestone in our quest to address the devastating impact of systemic sclerosis.”

The FDA’s Office of Orphan Products Development assigns orphan drug designation to novel drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect less than 200,000 people nationwide.