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Pompe Program Update

Author: 
Genzyme
Category: Archive
Published
December 18, 2004

Genzyme is still on track for the Marketing Authorisation Application (MAA) filing in December with the European regulatory authorities, and they will issue a corporate press release once the filing has been formally accepted. This will be a significant milestone, and we hope that this will be the first of many successful filings for regulatory approval of the investigational rhGAA enzyme. Genzyme anticipates that the European Medicines Authority (EMEA) will render a decision within 12-18 months of the filing, but this timeline is highly variable and is subject to change.

Genzyme also anticipates filing a Biologics License Agreement (BLA) with the US Food and Drug Administration (FDA) by the middle of 2005. Additional international filings will follow.

Overall, the last 6 months have been quite extraordinary for the Genzyme Pompe Program. Three clinical trials are fully enrolled, the Expanded Access Program has initiated approximately 40 new infantile and late-onset patients on investigational enzyme therapy, and we will continue to see patients treated and enrolled in this program.

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