GENZYME CORPORATION 
By Based on the global clinical experience of nearly 900 patients of all ages currently receiving Myozyme produced at the larger scale, including patients who participated in the Late-Onset Treatment Study (LOTS), Genzyme believes that Myozyme produced at both the 160L and 2000L scales is clinically effective and safe. Myozyme is the only treatment for Pompe disease -- a severe, progressively debilitating and life-threatening inherited disorder affecting a very small number of people throughout the world.
The FDA will require Genzyme to submit a separate biologics license
application (BLA) to gain approval for Myozyme produced at the 2000L scale.
The agency proposed that Genzyme initiate a rolling BLA review process by
submitting results from the LOTS study. Genzyme expects the FDA to give the
BLA priority review and to act on the application by the end of this year.
The LOTS study, which met its co-primary efficacy endpoints, was undertaken to
evaluate the safety and efficacy of Myozyme in juvenile and adult patients
with Pompe disease. Genzyme had already been preparing to submit results from
this study to the FDA to fulfill a post-marketing commitment. Genzyme
anticipates that this process will culminate in the availability of two
commercial versions of Myozyme in
To ensure that severely affected adults with Pompe disease in
"We are extremely disappointed in the FDA's decision because it will
further delay broad patient access to Myozyme, which is not possible under the
MTAP program," said
Financial Impact
Myozyme sales for 2008 are now expected to be approximately
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
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 regarding Genzyme's financial outlook and business plans and strategies, including without limitation: its 2008 earnings guidance; its 2008 Myozyme revenue guidance; its plans regarding the timing and content of the BLA submission for Myozyme manufactured at the 2000L scale; it expectations regarding FDA approval of that BLA and the timing thereof; its anticipated non-GAAP compound growth rate through 2011; and its belief there is no difference in the clinical effectiveness or safety of Myozyme produced at the 160L and the 2000L scales. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those forecasted. These risks and uncertainties include, among others: Genzyme's ability to obtain and maintain regulatory approvals for Myozyme produced at the 2000L scale and the timing thereof; Genzyme's ability to accurately anticipate regulatory actions on its Myozyme manufacturing applications; Genzyme's ability to manufacture its products, including Myozyme, in a timely and cost effective manner and in sufficient quantities to meet demand; Genzyme's ability to accurately forecast Myozyme revenues and the impact of reduced Myozyme revenues and other costs associated with the regulatory delay on earnings; and the risks and uncertainties described in Genzyme's SEC reports filed under the Securities Exchange Act of 1934, including the factors discussed under the caption "Risk Factors" in Genzyme's 2007 Annual Report on Form 10K. Genzyme cautions investors not to place substantial reliance on the forward-looking statements contained in this press release. These statements speak only as of today's date and Genzyme undertakes no obligation to update or revise the statements.
Genzyme(R) and Myozyme(R) are registered trademarks Genzyme Corp. All rights reserved.
Conference Call Information
Genzyme will host a conference call today at
Genzyme's press releases and other company information are available at
www.genzyme.com and by calling Genzyme's investor information line at 1-800-
905-4369 within
Media Contact:
Bo Piela
(617) 768-6579
Investor Contact:
Patrick Flanigan
(617) 768-6563
SOURCE Genzyme Corporation
