Proteolix's Carfilzomib granted orphan drug designation

Apr 3 2008

Proteolix, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to carfilzomib, a selective blocker of proteasome activity for the treatment of multiple myeloma.

The orphan drug designation encompasses all subsets of multiple myeloma and Waldenstrom's macroglobulinemia.

"I am very pleased by our receipt of this designation for carfilzomib," said Susan Molineaux, Ph.D., the Company's President and CEO. "To date we have been encouraged by carfilzomib's early-stage clinical results in multiple myeloma, and we continue to believe it has the potential to offer cancer patients a new and effective treatment for their disease."

The Orphan Drug Act of 1983 allows the FDA to grant orphan drug status to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. According to the SEER Database of the National Cancer Institute (2004), the U.S. prevalence of multiple myeloma was 53,712 patients. The Orphan Drug Act provides the drug developer with several financial benefits and incentives related to the orphan drug, including tax credits for clinical research costs, ability to apply for annual grant funding, clinical research trial design assistance, waiver of Prescription Drug User Fee Act (PDUFA) filing fees, and a seven-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication.

Multiple myeloma is cancer of the plasma cell. It begins in the bone marrow, where blood cells are made. The plasma cell is a type of white blood cell that fights infections by making antibodies. Cancerous plasma cells, also called myeloma cells, divide and grow out of control, building up in the marrow and crowding out healthy blood cells. The myeloma cells do not make effective antibodies and instead release abnormal proteins, called M proteins. M proteins can damage the immediate area and travel through the bloodstream and damage other parts of the body. Left untreated, multiple myeloma can cause bone damage, elevated blood calcium, anemia, and predisposition to infection and kidney damage. In 2007, there were an estimated 19,900 new cases diagnosed and 10,790 deaths due to multiple myeloma in the United States. For more information on multiple myeloma go to http://www.cancer.gov/cancertopics/types/myeloma.

Waldenstrom's Macroglobulinemia (WM) is a slow-growing type of non-Hodgkin's lymphoma that starts in white blood cells called B lymphocytes. WM is also called lymphoplasmacytic lymphoma because it occurs when abnormal lymphoplasmacytic cells multiply out of control, producing large amounts of a protein called monoclonal immunoglobulin M. WM can result in enlargement of the liver and spleen due to anemia and large amounts of immunoglobulin M can lead to hyperviscosity syndrome, in which the blood becomes abnormally thick. Symptoms of this syndrome include visual problems such as blurring or loss of vision and neurological problems including headache, dizziness, or vertigo. WM is a rare cancer with only about 1,500 new cases diagnosed annually in the United States. For more information about WM, go to http://www.cancer.gov/cancertopics/factsheet/Sites-Types/WM.