Genzyme Study of Myozyme(R) for Late-Onset Pompe Patients Meets Co-Primary Efficacy...(ectively defined subgroups. These ex...)

"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," s
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