CAMBRIDGE, Mass., Dec. 13 /PRNewswire-FirstCall/ -- Genzyme Corp.
(Nasdaq: GENZ) announced today that its Late Onset Treatment Study (LOTS)
of Myozyme(R) (alglucosidase alfa) met its co-primary efficacy endpoints.
The study was undertaken to evaluate the safety and efficacy of Myozyme in
juvenile and adult patients with Pompe disease. Myozyme was first approved
in 2006, and the product is now registered in 36 countries.
The randomized, double-blind, placebo-controlled study enrolled 90
patients at eight primary sites in the United States and Europe.
Participants received either Myozyme or a placebo every other week for 18
months. The average age of study participants was 44 years. The primary
efficacy endpoints of the study sought to determine the effect of Myozyme
on functional endurance as measured by the six-minute walk test and to
determine the effect of Myozyme on pulmonary function as measured by
percent predicted forced vital capacity.
The results showed that, at 18 months, patients treated with Myozyme
increased their distance walked in six minutes by an average of
approximately 30 meters as compared with the placebo group (P=0.0283;
Wilcoxon test). The placebo group did not show any improvement from
baseline. The average baseline distance walked in six minutes in both
groups was approximately 325 meters.
Percent predicted forced vital capacity in the group of patients
treated with Myozyme increased by 1 percent at 18 months. In contrast, it
declined by approximately 3 percent in the placebo group (P=0.0026;
Wilcoxon test). The average baseline percent predicted forced vital
capacity in both groups was approximately 53 percent.
The results for both efficacy endpoints were consistent across various
prospectively defined subgroups.
"These exciting findings build on our pivotal study results and
underscore the benefit of Myozyme to all patients with Pompe disease, said
Richard A. Moscicki, senior vice president and chief medical officer for
Genzyme. "The preservation of pulmonary function is extremely important
because respiratory failure is the major cause of mortality in this
progressive and life- threatening neuromuscular disease. This trial has
broader implications, in that it is one of the first large randomized
trials to show benefit in a degenerative neuromuscular disease. On behalf
of Genzyme, I want to thank the patients and physicians who participated in
this study, whose commitment to ensuring its successful completion was a
service to the entire Pompe community."
The safety of Myozyme was similar to placebo in the LOTS study. The
number of patients with serious and treatment-emergent non-serious adverse
events was similar in the Myozyme and placebo groups. Approximately 25
percent of patients in each group experienced infusion-associated
reactions. There was one death in the Myozyme group unrelated to treatment.
Genzyme is completing an analysis of the study results and will apply
in the second half of next year for potential inclusion of the results in
the product labeling. Detailed results will be presented at medical
congresses throughout the world by the study investigators and submitted
for publication in a peer-reviewed journal.
Myozyme used in the LOTS study was produced at Genzyme's Allston
Landing facility using the larger scale manufacturing process (2000L) that
is currently approved by 35 countries. The FDA is currently reviewing
Genzyme's application for approval of this larger scale process.
"This is a real breakthrough for the treatment of Pompe patients, as
for the first time it has been demonstrated in a large placebo-controlled
trial that Myozyme elicits a positive effect in adults with Pompe disease,"
said Ans van der Ploeg, M.D., Ph.D., of Erasmus Medical Center in
Rotterdam, one of the study's principle investigators. "This confirms
previous benefits reported in individual patients by our group, as well as
others, and extends the proven benefits across the spectrum of the
disease."
About Pompe Disease
Pompe disease manifests as a broad spectrum of clinical symptoms. All
patients typically experience progressive muscle weakness and breathing
difficulty, but the rate of disease progression can vary widely depending
on the age of onset and the extent of organ involvement. When symptoms
appear within a few months of birth, babies frequently display a markedly
enlarged heart and die within the first year of life. When symptoms appear
during childhood, adolescence or adulthood, patients may experience
steadily progressive debilitation and premature mortality due to
respiratory failure. They often require mechanical ventilation to assist
with breathing and wheelchairs to assist with mobility.
About Myozyme
Myozyme (alglucosidase alfa) is indicated for use in patients with
Pompe disease (GAA deficiency). The U.S. product label includes a boxed
warning with information on the potential risk of hypersensitivity
reaction. Life- threatening anaphylactic reactions, including anaphylactic
shock, have been observed in patients during Myozyme infusion. Because of
the potential for severe infusion reactions, appropriate medical support
measures should be readily available when Myozyme is administered. Full
prescribing information for the product, including a complete list of the
most common adverse reactions, is available on Genzyme's website:
http://www.genzyme.com/components/highlights/mz_pi.pdf
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 2006 revenues of $3.2 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.
In 2006 and 2007, Genzyme was selected by FORTUNE as one of the "100 Best
Companies to Work for" in the United States.
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.
This press release contains forward-looking statements, including
statements regarding anticipated regulatory submissions, the potential
inclusion of study data in product labeling and plans to present and
publish study results. These risks and uncertainties include, among others,
the actual timing of the completion of the analysis of study results; the
timing and content of submissions to and decisions by regulatory
authorities related the product labeling for Myozyme; and the risks and
uncertainties described in reports filed by Genzyme with the U.S.
Securities and Exchange Commission, including without limitation the
factors discussed under the caption "Risk Factors" in Genzyme's Quarterly
Report on Form 10-Q for the quarter ended September 30, 2007. We caution
investors not to place undue reliance on the forward-looking statements
contained in this press release. These statements speak only as of the date
of this press release, and we undertake no obligation to update or revise
the statements.
Genzyme(R) and Myozyme(R) are registered trademarks of Genzyme
Corporation. All rights reserved.
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: Investor Contact:
Bo Piela Sally Curley
(617) 768-6579 (617) 768-6140
SOURCE Genzyme Corp.
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CONTACT:
Media, Bo Piela, +1-617-768-6579, or
Investors, Sally Curley, +1-617-768-6140, both of Genzyme Corp.
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