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FDA Grants Orphan Drug Designation to BAY 43-9006 for the Treatment of Renal Cell Carcinoma

WEST HAVEN, Conn. and RICHMOND, Calif., Oct. 27 /PRNewswire-FirstCall/ -- Bayer Pharmaceuticals Corporation and Onyx Pharmaceuticals, Inc. announced today that sorafenib (BAY 43-9006) has been granted orphan drug status for the treatment of renal cell carcinoma by the U.S. Food and Drug Administration (FDA). The compound is being evaluated for the treatment of metastatic renal cell carcinoma, an advanced form of kidney cancer. A similar designation has been granted in the European Union by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA). Currently in Phase III clinical testing, BAY 43-9006 is a novel RAF kinase and VEGFR inhibitor that is intended to prevent tumor growth by combining two anticancer activities: inhibition of tumor cell proliferation and tumor angiogenesis. BAY 43-9006 is being co-developed by Bayer and Onyx.

The FDA orphan drug designation provides incentives to companies that develop drugs for diseases affecting less than 200,000 people in the United States.

"The orphan drug designation in the U.S. signifies another important step for Bayer and Onyx in the development of BAY 43-9006," said Susan Kelley, M.D., vice president, Oncology, Bayer Pharmaceuticals Corporation. BAY 43-9006 recently received the FDA's fast track designation and both the fast track and orphan drug designation provide mechanisms for more frequent communications with the FDA, helping to streamline development as well as the review and approval process.

BAY 43-9006, a novel investigational drug candidate, has demonstrated both anti-proliferative and anti-angiogenic properties -- two important anticancer activities. In preclinical models, BAY 43-9006 inhibited tumor cell proliferation by targeting the RAF/MEK/ERK signaling pathway at the level of RAF kinase. BAY 43-9006 also exerted an antiangiogenic effect by targeting the receptor tyrosine kinases VEGFR-2 and PDGFR and their associated signaling cascades.

Renal cell carcinoma is the most common form of kidney cancer. Nearly 190,000 people worldwide (about 32,000 Americans) are diagnosed with renal cell carcinoma each year, and more than 91,000 of them (about 12,000 Americans) die from the disease annually.

About Orphan Drug Designation

In the U.S., orphan drug designation provides a drug U.S. market exclusivity for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee. The designation does not shorten the duration of the regulatory review and approval process. If a product that has orphan drug designation is the first such product to receive FDA approval for the disease/indication for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same drug in the U.S. for the same disease, except in limited circumstances, for seven years. The FDA may permit additional companies to market the same drug for the designated condition if such companies can demonstrate better safety, efficacy, or a major contribution to patient care. The FDA may also approve more than one product for the same orphan indication or disease as long as the products are different drugs.

Source: Bayer Pharmaceuticals Corporation; Onyx Pharmaceuticals, Inc.

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