AnorMED has filed an orphan drug application for AMD-3100, a potential new agent for stem cell transplantation in cancer patients, with the U.S. Food and Drug Administration. Orphan drug status entitles a company to various incentives including the wavier 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 or biological product 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.
"We believe AMD-3100 has a potential role to play in improving the treatment of cancer patients undergoing the stem cell transplantation procedure," said Dr. Michael Abrams, CEO and President at AnorMED. He added, "The orphan drug designation offers important financial and regulatory benefits that will help us in the clinical and commercial development of this drug candidate".
AMD-3100 is a novel drug candidate, developed by AnorMED, that blocks a specific cellular receptor triggering the movement of stem cells out of the bone marrow and into circulating blood. Once in the circulating blood these stem cells can be collected for transplantation. The ability to collect as many stem cells as possible from the patient prior to their cancer treatment can improve the chances that their immune system can be restored. AMD-3100 is currently being evaluated in a Phase II clinical trial for its potential to improve the procedure and outcome of stem cell transplantation in cancer patients.
AnorMED's core strength involves the application of chemistry, biochemistry and biology to the discovery and development of small molecule therapeutics for the treatment of diseases including HIV, rheumatoid arthritis, asthma and cancer. The Company's clinical products include: FOSRENOL, exclusively licensed to Shire Pharmaceuticals Group plc, for which subject to regulatory approval, a launch is planned for 2003; AMD-3100, a potential new agent for stem cell transplant in cancer patients currently in Phase II trials; AMD-070 a new HIV entry inhibitor drug candidate expected to start clinical trials in 2003; AMD-473, a new platinum based anti-cancer agent that has completed a number of Phase I and Phase II trials; and HYNIC linker technology, a diagnostic and prognostic medical imaging technology licensed, non-exclusively, to North American Scientific, Inc. and to Bristol-Myers Squibb Medical Imaging Inc.
AnorMED's research is focused on a novel class of compounds that target specific chemokine receptors known to be involved in a variety of diseases. AnorMED continues to build upon its drug discovery and clinical development capabilities to support the rapid advancement of its most promising therapeutic candidates into clinical trials and to attract corporate partners.