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FDA and Genzyme Announce Label Expansion of Lumizyme in United States

Posted on: August 01, 2014

To the United States Pompe Community:

On August 1, 2014 the US Food and Drug Administration (FDA) and Genzyme issued press releases regarding the expansion of the Lumizyme® label (the enzyme replacement therapy for Pompe disease produced at the 4000L manufacturing scale).

The practical effect of the label expansion is that all patients in the United States will now be able to receive Lumizyme┬« through commercial therapy. Prior to this label expansion, Lumizyme┬« was only commercially available to Pompe patients over the age of eight (8). 

This label expansion only affects patients in the United States because it is only here that Genzyme has had two products available for the treatment of Pompe disease. All Pompe patients outside of the United States receive Myozyme produced at the 4000L manufacturing scale, which is comparable to Lumizyme in the United States.

Please see below for the Press Releases from the FDA and Genzyme, as well as a Pompe Program Update regarding the label expansion from Genzyme.

If you have any questions, please contact me at:

Thank you.

Tiffany House
AMDA President

FDA Press Release:
Genzyme Press Release
Genzyme Pompe Community Update

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San Antonio, Texas 78270 USA