CAMBRIDGE, Mass., June 17 /PRNewswire-FirstCall/ -- Genzyme Corporation
(Nasdaq: GENZ) announced today that it has submitted marketing applications
in both the United States and the European Union for Mozobil(TM)
(plerixafor), a product candidate intended to enhance mobilization of
hematopoietic stem cells for collection and subsequent autologous
transplantation in patients with lymphoma and multiple myeloma. The company
has requested priority review of its U.S. application and, if granted,
Mozobil could be approved by the end of this year. European approval is
expected in 2009. Additional global applications in up to 60 countries are
expected to follow.
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-Hodgkin's and Hodgkin's 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.
Commercialization Plans
Genzyme plans to launch Mozobil in the U.S. and Europe in 2009. Upon
commercial launch, Mozobil will be marketed and sold by Genzyme's 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, Hodgkin's 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 Mozobil's 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.
About Mozobil
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.
About Genzyme
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.
Genzyme(R) and AnorMED(R) are registered trademarks of Genzyme
Corporation or its subsidiaries. Mozobil(TM) is an unregistered trademark
of Genzyme Corporation or its subsidiaries. All rights reserved.
This press release contains forward-looking statements, including the
statements regarding: the anticipated timing of product approvals in the
U.S. and Europe, the anticipated timing of filing additional applications
for approval in other countries, the potential adoption of Mozobil in the
marketplace, the potential benefits to different types of patients,
commercial launch plans and Mozobil's potential use in other therapeutic
applications. These statements are subject to risks and uncertainties that
could cause actual results to differ materially from those projected in
these forward- looking statements. These risks and uncertainties include,
among others, the failure to receive regulatory approval to commercialize
Mozobil in the U.S., Europe or other countries, the lack of adoption of the
product in other therapeutic areas, the inability to change current
treatment protocols and risks associated with commercial launch and the
risks and uncertainties described in reports filed by Genzyme with the
Securities and Exchange Commission under the Securities Exchange Act of
1934, as amended, including without limitation the information under the
heading "Risk Factors" in the Management's Discussion and Analysis of
Financial Condition and Results of Operations section of the Genzyme
Quarterly Report on Form 10-Q for the quarter ending March 31, 2008.
Genzyme cautions investors not to place substantial reliance on the
forward-looking statements contained in this press release. These
statements speak only as of the date of this press release, and Genzyme
undertakes no obligation to update or revise the statements.
Genzyme's press releases and other company information are available at
http://www.genzyme.com and by calling Genzyme's investor information line at
1-800- 905-4369 within the United States or 1-678-999-4572 outside the
United States.
Media Contact:
Bo Piela
(617) 768-6579
Investor Contact:
Patrick Flanigan
(617) 768-6563
SOURCE Genzyme Corporation
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http://www.genzyme.com
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CONTACT:
Media, Bo Piela, +1-617-768-6579; or
Investors, Patrick Flanigan, +1-617-768-6563
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