Genzyme Receives FDA Approval for Mozobil

Press Release, December 15, 2008

The biotech company Genzyme has received approval from the FDA for the marketing of its proprietary adult stem cell stimulating product, Mozobil, which is a novel, proprietary small-molecule CXCR4 chemokine receptor agonist. When used in combination with G-CSF (granulocyte-colony stimulating factor), Mozobil has been shown to increase the number of stem cells circulating in the peripheral blood by mobilizing hematopoietic stem cells in the bone marrow and stimulating the cells to migrate into the bloodstream for easier collection and subsequent autologous transplantation.

According to John DiPersio, M.D., Ph.D., a professor at Washington University in St. Louis, "Mozobil is an important advancement in the treatment of patients with certain types of cancer who require a stem cell transplant. This product should become an integral part of the treatment regimen for transplantation because of the benefits it offers to patients, physicians and transplant centers."

In order to receive an autologous (in which the donor and recipient are the same person) adult stem cell transplant, each patient must be able to donate a minimum of 2 million stem cells per kilogram of body weight. After being collected, isolated and expanded, the adult stem cells are then administered to the patient as part of the therapy. For some patients, the collection process alone can take hours, spread out over multiple days, and for those patients who are not able to mobilize enough stem cells for the collection, the transplant is not possible. By mobilizing a patient's endogenous stem cells and by stimulating the stem cells to migrate from the bone marrow into the bloodstream, Mozobil facilitates the therapeutic adult stem cell process, especially for patients who might not otherwise be able to receive such a therapy at all.

According to Joseph Lobacki, senior vice president and general manager of Genzyme's transplant and oncology division, "Mozobil is an exciting and innovative new treatment that expands Genzyme's contribution to the field of hematology and oncology. We look forward to strengthening our partnership with the blood and marrow transplant community to make this product broadly available to patients who are facing transplantation procedures for non-Hodgkin's lymphoma or multiple myeloma."

Approximately 55,000 hematopoietic stem cell transplants are performed each year globally for multiple myeloma, Hodgkin's and non-Hodgkin's lymphoma, for which Mozobil is expected to be used in the majority of such cases. Peak annual sales of Mozobil are projected to reach $400 million. Genzyme has also submitted applications to the respective regulatory agencies in Europe, Australia and Brazil for marketing approval of Mozobil, and additional applications in up to 60 countries are planned.

Founded in 1981, Genzyme now has more than 10,000 employees around the world with revenues in 2007 of $3.8 billion. With products and services available in nearly 90 countries worldwide, Genzyme focuses on the treatment of rare inherited disorders, kidney disease, orthopedics, cancer, transplantation, and diagnostic testing. In 2007, Genzyme was awarded the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.