Pompe Program Update

Category: Archive
March 20, 2005

Continued progress is being made in the Pompe program at Genzyme, with the goal of obtaining worldwide regulatory approval for the investigational rhGAA enzyme. There are now over 110 patients receiving investigational rhGAA, and as a result, there are increasing numbers of physicians and hospitals around the world having direct experience with the investigational enzyme.

The submission (MAA) to the European regulatory authorities (EMEA) was formally accepted in December 2004. The regulatory submission to the FDA in the US (BLA) is still on track for mid-2005, with additional international filings to follow. Consultations with regulatory authorities are ongoing, in regards to these submissions and the upcoming Late-Onset Treatment Study. We hope and expect that much progress will be made in 2005.

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