Two-Year Data from Phase 2 Trial of Genzyme’s Eliglustat Tartrate for Gaucher Disease To Be Published in the Journal Blood

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme Corporation (NASDAQ: GENZ) announced today that the two-year follow-up results from the phase 2 clinical trial of its investigational therapy known as eliglustat tartrate have been accepted for publication in the journal Blood. The results have been pre-published on the journal’s website and are available to subscribers.

Eliglustat tartrate, a capsule taken orally, is being developed to provide a convenient treatment alternative for adult patients with Gaucher disease type 1, and to offer a broader range of treatment options for patients and physicians to achieve individual therapeutic goals. Current treatments, including Genzyme’s Cerezyme® (imiglucerase for injection), the standard of care for patients with Gaucher disease type 1, are administered through intravenous infusions.

Genzyme reported last year that the 52-week phase 2 trial of eliglustat tartrate had met its primary composite endpoint: a clinically meaningful response in at least two of three endpoints (improvements in spleen size, hemoglobin and platelet levels) in individual patients. The two-year data to be published in Blood indicate continued improvements across all endpoints. After two years, most patients (85%) had met at least three of the four hematologic and visceral therapeutic goals established for enzyme replacement therapy. These data also suggest that eliglustat tartrate may positively impact two indicators of bone disease, bone mineral density and the presence of dark marrow.

The most common adverse events reported in greater than 10 percent of patients included viral infections (six patients), urinary tract infections, increased blood pressure, and abdominal pain (three patients each). Eight drug-related adverse events, including one serious event, were reported in six patients. All were mild in severity.

These two-year results were presented at the Lysosomal Disease Network WORLD Symposium earlier this year.

Phase 3 Program

Genzyme is currently enrolling patients in three global, multi-center, phase 3 trials of eliglustat tartrate. Combined, more than 450 patients are expected to participate, making this the largest clinical program ever focused on Gaucher disease. Over 50 sites in more than 25 countries are currently participating, with additional centers planned.

The first phase 3 trial, ENCORE, is a randomized, open-label study for adult patients with Gaucher disease type 1, designed to compare eliglustat tartrate to Cerezyme. Adult patients who have previously received enzyme replacement therapy for at least three years and have reached their therapeutic goals may qualify for this trial. The second trial, ENGAGE, is a randomized, double-blind, placebo-controlled study for patients with a confirmed diagnosis of Gaucher disease type 1. Patients who have not received enzyme replacement therapy within 9 months or substrate reduction therapy within 6 months of participation may qualify for this study. Genzyme recently launched a third trial, known as EDGE, to compare once-daily dosing of eliglustat tartrate with twice-daily dosing.

“We remain very enthusiastic about the potential of eliglustat tartrate,” said Genzyme’s President of Personalized Genetic Health, John Butler. “Through this robust phase 3 program, we look forward to increasing our understanding of the compound as we work to offer patients and physicians more treatment choices to help optimize the management of Gaucher disease.”

Complete results from the one-year analysis period for eliglustat tartrate were previously published in Blood. The study and accompanying commentary can be found here.

To learn more about enrolling in the Phase 3 trials, contact Genzyme Medical Information at or 1-800-745-4447. More information can also be found at

About Gaucher disease

Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide. People with Gaucher disease do not have enough of an enzyme, β-glucosidase (glucocerebrosidase) that breaks down a certain type of fat molecule. As a result, lipid engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver and bone marrow. Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease and a number of other signs and symptoms. The most common form of Gaucher disease, type 1, does not affect the brain or nervous system.

About eliglustat tartrate

Eliglustat tartrate, a novel glucosylceramide analog given orally, is designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide is the substance that builds up in the cells and tissues of people with Gaucher disease. In preclinical studies, the molecule, developed with James A. Shayman, MD, from the University of Michigan, has shown high potency and specificity. Based on its mechanism of action, which is independent of genotype, eliglustat tartrate may be a potential therapy for all patients with Gaucher disease type 1. Initiation of the Phase 2 and 3 studies of eliglustat tartrate in Gaucher disease followed completion of an extensive pre-clinical research effort and a Phase 1 program that involved more than 120 subjects in three separate studies. The most common adverse events (AEs) by two years included viral infections, urinary tract infections, increased blood pressure and abdominal pain.

Cerezyme important safety information

Approximately 15 percent of patients have developed IgG antibodies. These patients have a higher risk of hypersensitivity reaction. Therefore periodic monitoring is suggested; caution should be exercised in patients with antibodies or prior symptoms of hypersensitivity. Symptoms suggestive of hypersensitivity occurred in 6.6 percent of patients, and include anaphylactoid reaction, pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, coughing, cyanosis and hypotension. Reactions related to Cerezyme administration have been reported in less than 15 percent of patients. Each of the following events occurred in less than two percent of the total patient population. Reported adverse events include nausea, vomiting, abdominal pain, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache and tachycardia. Adverse events associated with the route of administration include discomfort, pruritus, burning, swelling or sterile abscess at the site of venipuncture. For full prescribing information, please visit

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 12,000 employees in locations spanning the globe and 2009 revenues of $4.5 billion. In 2010, Genzyme was named to the Fortune 500.

With many established products and services helping patients in 100 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 immune disease. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as cardiovascular disease, neurodegenerative diseases, and other areas of unmet medical need.

Genzyme’s press releases and other company information are available at 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.

Genzyme® and Cerezyme® are registered trademarks of Genzyme Corporation. All rights reserved.

This press release contains forward looking statements regarding Genzyme’s investigational therapy eliglustat tartrate including: that eliglustat tartrate will offer a broad range of treatment options to physicians and convenience to patients and that eliglustat tartrate could have a significant impact on bone mineral density and make a positive impact on patients’ lives. These forward looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those forecasted. These risks and uncertainties include: that regulatory approval of eliglustat tartrate will be delayed or limited either in the United States or elsewhere, that eliglustat tartrate will not be available for patients’ convenience and will not provide additional treatment options to physicians or patients; that eliglustat tartrate will not have a positive effect on bone mineral density or otherwise on bone disease and will not impact patients lives; 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 Genzyme’s Quarterly Report on Form 10-Q for the quarter ending June 30, 2010. 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 these statements.

Genzyme Corporation
Lori Gorski, 617-768-9344
Patrick Flanigan, 617-768-6563

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