Will Host Conference Call Today at 5:00 p.m.
CAMBRIDGE, Mass., April 21 /PRNewswire-FirstCall/ -- Genzyme
Corporation (Nasdaq: GENZ) announced today that the FDA has informed the
company of its opinion that Myozyme(R) (alglucosidase alfa) produced at the
160L bioreactor scale and Myozyme produced at the 2000L scale should be
classified as two different products because of differences in the
carbohydrate structures of the molecules. Currently, Genzyme has U.S.
approval to sell Myozyme manufactured at the 160L scale, and the company
has been seeking clearance from the FDA for Myozyme produced at the 2000L
scale. Production at this larger scale has already been approved in more
than 40 countries.
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 the United States: one produced at
the 160L scale and the other produced at the 2000L scale. The company
expects to begin providing U.S. patients with commercial 2000L Myozyme
during the first quarter of 2009.
To ensure that severely affected adults with Pompe disease in the
United States have access to treatment, Genzyme, in collaboration with the
FDA, created the Myozyme Temporary Access Program (MTAP) in May 2007.
Through this program the company is currently providing Myozyme produced at
the 2000L scale free of charge to approximately 140 patients. Infants and
children with Pompe disease in the United States continue to receive
commercially approved Myozyme produced at the 160L scale.
"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 Henri A. Termeer, Genzyme's chairman and chief
executive officer.
Financial Impact
Myozyme sales for 2008 are now expected to be approximately $275-$285
million compared to previous guidance of $320-$330 million, reflecting the
delay in the approval of 2000L production. Genzyme anticipates that this
delay will have an impact on 2008 non-GAAP earnings of approximately $0.10
per diluted share. This reflects both forgone commercial sales margin and
the costs of continued administration of the MTAP program. Genzyme now
expects 2008 non-GAAP earnings of approximately $3.90 per diluted share,
compared with previous guidance of $4.00 per diluted share. GAAP earnings
for 2008 are now expected to be approximately $2.65 per diluted share,
compared with previous guidance of $2.75 per diluted share. GAAP figures
include anticipated amortization and stock-compensation expenses and the
effect of contingent convertible debt. Genzyme reaffirmed its commitment to
20 percent compound average growth in non-GAAP earnings per share through
2011.
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.
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 5:00 p.m. Eastern to
discuss Myozyme manufacturing. To participate in the call, please dial
1-773-799-3828 and use passcode "Genzyme." The replay number for the call
is 203-369-3292, and the replay will be available until midnight on May
5th. Today's call will also be Webcast on the investor events section of
http://www.genzyme.com.
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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Related links:
http://www.genzyme.com
http://www.prnewswire.com/comp/113803.html /
CONTACT:
Media, Bo Piela, +1-617-768-6579, or
Investors, Patrick Flanigan, +1-617-768-6563, both of Genzyme
Corporation
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