PRIVIGEN Earns Orphan Drug Designation as a Treatment for Scleroderma

According to a story from Benzinga, the biotherapeutics company CSL Behring has recently announced that its medical product PRIVIGEN ® has earned Orphan Drug designation from the US Food and Drug Administration (FDA). This designation is for the use of PRIVIGEN as a treatment for scleroderma, a rare disease. PRIVIGEN is a ready-to-use formulation of intravenous immunoglobulin (IVIG). There are currently no disease modifying therapies approved by the FDA for this illness.

About Scleroderma

Scleroderma, which is also referred to as systemic sclerosis, describes a group of autoimmune diseases that can cause system-wide effects in the most severe cases. The mechanism of this disease is believed to be an autoimmune response in which the immune system mistakenly attacks body tissue. Some factors that may contribute to triggering the autoimmune response include mutations of the HLA genes and exposure to certain materials, such as certain solvents, white spirits, ketones, and silica. Symptoms are broad ranging and systemic, including kidney failure, erectile dysfunction, fatigue, stroke, headaches, facial pain, congestive heart failure, skin abnormalities, high blood pressure, chest pain, indigestion, and many more. Treatments are varied and depend on the symptoms, but most patients take medications in an attempt to suppress the autoimmune response. In severe cases, life expectancy is around 11 years from onset. To learn more about scleroderma, click here.

About Orphan Drug Designation

Orphan Drug designation is typically reserved for medications that are being developed as a treatment for a disease that is considered rare, which is defined as any condition that affects less than 200,000 people in the US. A drug may qualify for this designation if it fulfills a currently unmet medical need or appears to offer major advantages in efficacy or safety in comparison to current therapies. This designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug is approved.

CSL Behring has also received Fast Track designation for scleroderma. PRIVIGEN is currently approved as a treatment for several other rare diseases as well, including chronic inflammatory demyelinating polyneuropathy (CIDP), chronic immune thrombocytopenic purpura (ITP), and primary immunodeficiency (PI).

 


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