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Important Clinical Role
Mozobil is designed to mobilize stem cells from the bone marrow into thebloodstream where they can be collected, making it more likely for a patientwith certain types of cancers to receive a successful transplant.Specifically, patients with non-Hodgkin's and Hodgkin's lymphomas and multiplemyeloma often receive high-dose chemotherapy, a process that destroys bonemarrow. A stem cell transplant is required to replenish blood-forming bonemarrow cells destroyed by high-dose chemotherapy. Stem cells differentiateinto the mature red blood cells, white blood cells, and platelets that ahealthy person needs.
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Currently, before a transplant can take place, patients may receive aprescribed dose of chemotherapy and/or other drugs, called growth factors, tohelp mobilize their hematopoietic stem cells into the bloodstream. Once thecells are released into the bloodstream, they are easier to collect inpreparation for a transplant.
In order for the transplant to take place, a minimum number ofapproximately 2 million cells per kilogram of body weight must be collected.For many patients, this process can take three or four hours over multipledays to complete. Even then, some patients are not able to mobilize enoughcells, and a transplant is not possible.
Genzyme conducted two phase 3 studies that confirmed the potential ofMozobil to effectively and predictably prepare lymphoma and multiple myelomapatients for an autologous transplant. Both studies successfully met primaryand secondary endpoints. Patients who received Mozobil in conjunction with agrowth factor achieved more rapid and effective mobilization of stem cells inpreparation for autologous transplant than patients treated with growth factoralone. In addition, more patients treated with Mozobil plus a growth factorachieved a composite endpoint of optimal stem cell collection and successfultransplantation, compared to patients mobilized with placebo plus a growthfactor. Mozobil was well tolerated in both trials, with the most commonadverse 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 atGenzyme. "The product has great potential to meet an important, unmet medicalneed and has numerous potential benefits for patients."
More than 900 patients have received Mozobil through a compassionate useprogram in the United States, and similar compassionate use programs haverecently begun in Europe.
Commercialization Plans
Genzyme plans to launch Mozobil in the U.S. and Europe in 2009. Uponcommercial launch, Mozobil will be marketed and sold by Genzyme's existingTransplant sales force, which has a commercial presence in more than 55countries worldwide. In addition, the company will leverage its Oncologybusiness and clinical infrastructure.
Approximately 55,000 stem cell transplants are performed each year formultiple myeloma, Hodgkin's and non-Hodgkin's lymphoma, and other conditionsin markets where Genzyme has a commercial infrastructure, including the UnitedStates, Europe, Latin America and the Asian Pacific countries. Genzymebelieves that over time, Mozobil
