CSL Behring announced the U.S. Food and Drug Administration (FDA) has granted Privigen® (Immune Globulin Intravenous (Human), 10% Liquid) orphan-drug designation as an investigational therapy in the treatment of Systemic Sclerosis (SSc). SSc is a chronic and potentially life-threatening autoimmune disorder characterized by a build-up of scar tissue (fibrosis) in the skin and other organs. It affects approximately 100,000 people in the U.S. There are currently no FDA-approved, disease-modifying treatments to stop or reverse the overall course of the disease.
The FDA Office of Orphan Products Development (OOPD) grants orphan-drug designation to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 U.S. patients. The designation qualifies companies that invest in research and development of these medications for a range of incentives, including fee waivers, tax credits and the potential for marketing exclusivity upon approval.
"CSL Behring is driven by our promise to develop and deliver innovative therapies for patients with the highest unmet need," said Mittie Doyle, Vice President, Research and Development, Immunology and Neurology Therapeutic Area at CSL Behring. "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."
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Privigen is currently approved in the U.S. to treat patients with primary immunodeficiency (PI), chronic inflammatory demyelinating polyneuropathy (CIDP) and chronic immune thrombocytopenic purpura (ITP). CSL Behring recently initiated a Phase 2 clinical trial to evaluate the safety and efficacy of Privigen in the treatment of adults with SSc, and in October 2019 received Fast Track Designation for the clinical development of Privigen for the treatment of serious pulmonary, skin, and musculoskeletal manifestations resulting from this condition. The FDA confers Fast Track designation to aid and expedite the development and review of drugs that show promise in treating a serious or life-threatening disease and address an unmet medical need.
Systemic Sclerosis (SSc) is a rare, chronic, and potentially life-threatening autoimmune rheumatic disease characterized by the buildup of scar tissue or fibrosis resulting in hardening, thickening and tightening of the skin. In SSc, fibrosis can also affect internal organs, such as the esophagus, heart, lungs, and kidneys, often leading to their impairment or failure. SSc affects approximately 100,000 people in the United States, with females being four times more likely to develop the condition than males. Other factors, such as race and ethnicity, may also influence the risk of getting SSc, the age of onset and the pattern and severity of internal organ involvement.
Privigen® is the first and only 10 percent, ready-to-use, liquid IVIg stabilized with proline, a naturally occurring amino acid. Privigen was first approved in 2007 in U.S. for the treatment of patients with primary immunodeficiency (PI) and chronic immune thrombocytopenic purpura (ITP) in patients age 15 years and older. Since September 2017, Privigen has also been indicated for the maintenance treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) in adults and is available in more than 60 countries around the world to treat these and other rare diseases.