AnorMED Inc has received orphan drug designation for AMD-3100, a potential new agent in stem cell transplant in cancer patients from the US Food and Drug Administration (FDA). Orphan drug status entitles a company to various incentives including the waiver of regulatory filing fees, access to potential grant funding for non-clinical and clinical research undertaken to generate required data for marketing approval, and seven years of marketing exclusivity for a designated drug once approved by the FDA.

Stem cell transplantation involves the collection of certain types of stem cells that are used to restore the immune system of patients following their radiation and/or chemotherapy treatments for cancers involving the blood and immune system such as leukemias, multiple myeloma and lymphomas. It is estimated that in 2000 over 36,000 patients underwent stem cell transplant procedures in North America and Europe. This number is projected to grow to approximately 46,000 by 2005.

AMD-3100 is a novel drug candidate developed by AnorMED that blocks a specific cellular receptor triggering the rapid movement of stem cells out of the bone marrow and into circulating blood. Clinical data in healthy volunteers show that the combined use of AMD-3100 and Neupogen, the standard agent used in stem cell transplant, mobilizes a greater number of stem cells than either agent alone. Recent clinical data has also shown that AMD-3100, administered alone, mobilizes stem cells in cancer patients. AMD-3100, in combination with Neupogen, is currently in two Phase II clinical trials to evaluate its potential to increase the stem cells available for transplant, improve the transplantation procedure, and enhance patient outcome. The first Phase II trial is being conducted in multiple myeloma and non-hodgkins lymphoma patients, and the second Phase II trial in multiple myeloma patients who have previously failed to mobilize an adequate number of stem cells, referred to as poor mobilizers.