Genzyme Receives EU Approval for Adult Stem Cell Mobilization Product

The biotechnology giant Genzyme Corporation announced today that it has received regulatory approval from the European Commission for the commercial marketing of one of its products, Mozobil. A proprietary, injectable formulation of plerixa, Mozobil stimulates the mobilization and migration of the body’s own adult, hematopoietic stem cells out of the bone marrow and into the peripheral blood where the cells can be easily collected. The intended, initial use of Mozobil in the European market is for patients with lymphoma and multiple myeloma who require an autologous (in which the donor and recipient are the same person) adult stem cell transplant, but who are suspected of having inadequate mobilzation and who have previously failed conventional treatment.

According to Dr. Mohamad Mohty of the Stem Cell Transplant Program at the University Hospital in Nantes, France, Mozobil "has the potential to transform the field of stem cell transplantation. This new treatment will allow more patients with yet incurable malignancies to confidently move on to a potentially life-saving autologous stem cell transplant."

Mozobil has been evaluated in two randomized, double-blind, placebo controlled Phase III clinical trials as a treatment for patients with non-Hodgkin’s lymphoma and multiple myeloma. In addition to Mozobil, the patients were also given G-CSF (granulocyte-colony stimulating factor), a combination which proved to increase stem cell mobilization yield as well as graft durability rates as revealed in year-long follow-up data. Additionally, Mozobil has already been tested on more than 1,000 patients in Europe over the past year who received the treatment through "compassionate use" programs. Because it was formulated to treat specific medical conditions, Mozobil has been granted "orphan drug" status both in the United States and the European Union.

As a new option for a broad group of cancer patients, Mozobil offers distinct economic as well as clinical benefits. Since it has been found to decrease the number of days required for apheresis, Mozobil reduceds the need for a second mobilization procedure, thereby reducing the overall cost of the medical treatment. A minimum of approximately two million hematopoietic stem cells per kilogram of body weight must be collected before any patient can receive an autologous adult stem cell transplant, although twice that many stem cells would allow for a more effective outcome. Without Mozobil, many patients are unable to produce enough stem cells, even in multiple collection procedures that are performed over multiple days, in which case such patients are not allowed to proceed with the therapy. With Mozobil, however, most patients are able to produce an adequate amount of stem cells during the first collection procedure, and are therefore able to complete the entire transplant therapy in a more timely and efficient manner.

According to Paula Soteropoulos, Genzyme vice president and general manager of Mozobil, "There is strong interest among European physicians for Mozobil. With today’s approval, we will move quickly to make this important product broadly available to the transplant community."

Approximately 55,000 hematopoietic stem cell transplants are performed each year throughout the world for Hodgkin’s lymphoma, non-Hodgkin’s lymphoma, multiple myeloma and related disorders. Mozobil was approved for use in the U.S. in December of 2008, and in Mexico in May of 2009. In addition to the E.U., Genzyme has also filed applications for the approval of Mozobil in Argentina, Australia, Brazil, Israel and Singapore, with additional applications for other countries still in the planning stage. Global peak sales of Mozobil are projected to reach $400 million annually.

As the world’s third-largest biotechnology company, Genzyme currently employs more than 11,000 people around the world, in approximately 80 offices and laboratories that are located in 40 countries. In 2008, Genzyme reported $4.6 billion in global revenue from more than 25 products that are available throughout the world.

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