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FDA Grants Interleukin 21 Orphan Drug Status
"We're excited about the progress of the IL-21 program," said Dr. Bruce L.A. Carter, President and CEO of ZymoGenetics. "Our studies have shown that IL-21 stimulates cells of the immune system that are thought to have a critical role in eliminating malignant cells. We believe IL-21 may come to play an important role in helping cancer patients who need an effective therapy with manageable side effects."
About the FDA Orphan Drug Designation
The FDA Orphan Drug designation is reserved for promising new therapies being developed to treat diseases that affect fewer than 200,000 people in the United States. The Orphan Drug Act guarantees market exclusivity for seven years to the first sponsor that obtains market approval for an orphan-designated product. This designation provides companies with financial and regulatory benefits during the course of orphan drug development, including tax credits related to clinical trial expenses.
Melanoma is a form of skin cancer that spreads rapidly if not caught at an early stage. The American Cancer Society estimates that this year, nearly 60,000 Americans will be diagnosed with melanoma, and almost 8,000 patients will die from the disease. Nearly a quarter of melanoma patients have advanced or aggressive (Stages IIB-T4a and beyond) forms of the disease, and only thirty-nine percent of those patients are currently projected to survive beyond five years from the time of diagnosis. (Balch, C., et al; Final version of the American Joint Committee on Cancer Staging System for Cutaneous Melanoma. Journal of Clinical Oncology, Vol. 19, No. 16 (August 15), 2001: pp 3635-3648)
About Interleukin 21 (IL-21)
IL-21 is a novel cytokine with potent effects on a number of immune effector cells such as cytotoxic T cells and natural killer cells. These cell types are thought to play key roles in eliminating malignant and infected cells from the body.
Metastatic melanoma and renal cell carcinoma, two tumors which have been shown to be responsive to immune based therapies, were selected as the initial indications. In addition, IL-21 has been shown to inhibit tumor growth in animal models of melanoma and renal cell carcinoma with a mechanism involving regulation of immune cells. ZymoGenetics has retained the commercialization rights for IL-21 in North America and licensed the commercialization rights outside of North America to Novo Nordisk A/S.
Source: ZymoGenetics, Inc.