The US Food and Drug Administration (FDA) has granted orphan-drug status designation to the investigational drug Tiazole(TM) (tiazofurin) for the treatment of chronic myelogenous leukemia (CML) in accelerated phase or blast crisis.
Orphan-drug status designation is granted for rare diseases or conditions which affect fewer than 200,000 people in the United States. CML is not currently curable with conventional chemotherapy or immunotherapy. Such
status provides 7 years of marketing exclusivity after approval.
Discovered and developed by ICN Pharmaceuticals, Inc., the anti-cancer compound tiazofurin is currently being tested in patients with chronic myelogenous leukemia in accelerated phase or blast crisis, ovarian cancer, and multiple myeloma. In early studies, tiazofurin was found to have activity in patients with CML with blast crisis in clinical trials sponsored by the US National Cancer Institute (NCI).
Tiazofurin is currently in Phase II/III clinical trials. Upon successful
completion of required studies and tests, ICN intends to file a New Drug
Application (NDA) with the FDA for treatment of CML with blast crisis.
For more information, visit the website of ICN Pharmaceuticals at http://www.icnpharm.com