Important Clinical Role
Mozobil is designed to mobilize stem cells from the bone marrow into the bloodstream where they can be collected, making it more likely for a patient with certain types of cancers to receive a successful transplant. Specifically, patients with non-Hodgkinâs and Hodgkinâs lymphomas and multiple myeloma often receive high-dose chemotherapy, a process that destroys bone marrow. A stem cell transplant is required to replenish blood-forming bone marrow cells destroyed by high-dose chemotherapy. Stem cells differentiate into the mature red blood cells, white blood cells, and platelets that a healthy person needs.
Currently, before a transplant can take place, patients may receive a prescribed dose of chemotherapy and/or other drugs, called growth factors, to help mobilize their hematopoietic stem cells into the bloodstream. Once the cells are released into the bloodstream, they are easier to collect in preparation for a transplant.
In order for the transplant to take place, a minimum number of approximately 2 million cells per kilogram of body weight must be collected. For many patients, this process can take three or four hours over multiple days to complete. Even then, some patients are not able to mobilize enough cells, and a transplant is not possible.
Genzyme conducted two phase 3 studies that confirmed the potential of Mozobil to effectively and predictably prepare lymphoma and multiple myeloma patients for an autologous transplant. Both studies successfully met primary and secondary endpoints. Patients who received Mozobil in conjunction with a growth factor achieved more rapid and effective mobilization of stem cells in preparation for autologous transplant than patients treated with growth factor alone. In addition, more patients treated with Mozobil plus a growth factor achieved a composite endpoint of optimal stem cell collection and successful transplantation, compared to patients mobilized with placebo plus a growth factor. Mozobil was well tolerated in both trials, with the most common adverse events being gastrointestinal effects and injection site reactions.
"There is a lot of excitement among treating physicians about Mozobil," said Mark Goldberg, M.D., senior vice president of clinical research at Genzyme. "The product has great potential to meet an important, unmet medical need and has numerous potential benefits for patients."
More than 900 patients have received Mozobil through a compassionate use program in the United States, and similar compassionate use programs have recently begun in Europe.
Genzyme plans to launch Mozobil in the U.S. and Europe in 2009. Upon commercial launch, Mozobil will be marketed and sold by Genzymeâs existing Transplant sales force, which has a commercial presence in more than 55 countries worldwide. In addition, the company will leverage its Oncology business and clinical infrastructure.
Approximately 55,000 stem cell transplants are performed each year for multiple myeloma, Hodgkinâs and non-Hodgkin's lymphoma, and other conditions in markets where Genzyme has a commercial infrastructure, including the United States, Europe, Latin America and the Asian Pacific countries. Genzyme believes that over time, Mozobil will be used in the majority of these procedures, and peak sales of the product in the transplant setting are expected to reach $400 million annually.
In addition to the patient benefit, Mozobil also may offer significant economic benefits for transplant centers. Mozobil has the potential to decrease the number of apheresis days and provide transplant centers with more predictable and efficient use of the apheresis center, while reducing the number of patients who may fail to mobilize sufficient numbers of cells and therefore require a second mobilization procedure.
Additional Therapeutic Opportunities
Numerous Genzyme and investigator-sponsored trials are planned or underway to study Mozobilâs use in other settings such as allogeneic hematopoietic stem cell transplants. Genzyme is also studying the use of Mozobil to improve the efficacy of chemotherapy and/or immunotherapy in various types of hematologic malignancies such as chronic lymphocytic leukemia and acute myelogenous leukemia, and is pursuing preclinical work to explore the role that Mozobil may play in cord blood transplantation, solid organ transplantation, cardiovascular disease, renal ischemic disease, and a variety of additional types of solid tumor malignancies.
"We're just beginning to unlock the potential of Mozobil," said Joseph Lobacki, senior vice president of the Transplant business unit at Genzyme.
Mozobil, a novel small molecule CXCR4 chemokine receptor antagonist, has been shown in multiple earlier studies to rapidly and effectively increase the number of stem cells in circulation in the blood. Once circulating in the blood, stem cells can be collected for use in a stem cell transplant. Mozobil has been granted orphan drug status in the United States and European Union and the pivotal trials have undergone Special Protocol Assessment by the FDA and Protocol Assistance by the EMEA. Genzyme has been developing Mozobil since its acquisition of AnorMED, Inc. in 2006.
One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 10,000 employees in locations spanning the globe and 2007 revenues of $3.8 billion. In 2007, Genzyme was chosen to receive the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.
With many established products and services helping patients in nearly 90 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as immune disease, infectious disease, and other areas of unmet medical need.
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