You are here
Analog of Geldanamycin Granted Orphan Drug Status
"The orphan drug designation in the U.S. is an important milestone for the NCI and Kosan programs," said Robert G. Johnson, Jr., M.D., Ph.D., Executive Vice President, Development and Chief Medical Officer, Kosan Biosciences. "We expect this designation to provide Kosan with a competitive advantage for this specific oncology indication."
"Although there have been recent pharmacological advances in the treatment of multiple myeloma, a significant number of patients have a low likelihood of cure. 17-AAG, directed against a novel target in this disease, has shown remarkable anti-myeloma effects in our preclinical models; we hope that we see similar results in our patients," remarked Dr. Paul Richardson of the Dana Farber Cancer Institute.
17-AAG is also being evaluated for the treatment of multiple cancer indications in Phase II, Phase I, and Phase Ib studies in collaboration with the National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA) between Kosan and the NCI Cancer Therapy Evaluation Program (CTEP).
17-AAG inhibits Hsp90 (heat shock protein 90), a protein chaperone that binds to signaling proteins, known as "client proteins." These client proteins include a "who's who" list of cancer-relevant targets such as mutated p53, Bcr-Abl, Her2, Akt, Raf-1, B-Raf, and others. When 17-AAG binds to Hsp90, it disrupts the Hsp90-client protein complexes, leading to degradation of the client proteins.
Multiple myeloma (also known as myeloma or plasma cell myeloma) is a hematologic cancer that develops in the blood. It is a cancer of the plasma cell, an important part of the immune system that produces immunoglobulins (antibodies) to help fight infection and disease. There are approximately 45,000 people in the United States living with multiple myeloma and an estimated 14,600 new cases of multiple myeloma are diagnosed each year.
About Orphan Drug Designation
Orphan drug designation is granted by the U.S. Food and Drug Administration to products that treat rare diseases or conditions that affect fewer than 200,000 people in the United States. The designation provides eligibility for a special seven-year period of market exclusivity after marketing approval, potential tax credits for research, grant funding for research and development, possibly reduced filing fees for marketing applications, and assistance with the review of clinical trial protocols.
Source: Kosan Biosciences Incorporated