We would like to take this opportunity to provide an important update to the Pompe Community.
Background
In the early stages of development of Myozyme® (alglucosidase alfa), Genzyme initiated manufacturing at the 160 liter (L) scale, and has since scaled-up the process to produce larger quantities of product for commercial use worldwide. The bioreactors in which alglucosidase alfa has been produced over the course of time have steadily grown in size to the 2000 L scale that currently produces product for worldwide use including US patients enrolled in the Myozyme Temporary Access Program (MTAP). In the US, patients under 18 years of age continue to receive Myozyme produced at the 160 L scale. This incremental scale-up is standard manufacturing practice for any biological drug development process. In the US, the FDA determined that alglucosidase alfa produced at the 160 L scale and alglucosidase alfa produced at the 2000 L scale should be classified as two different products. A new Biologics License Application (BLA) was submitted to the FDA for the 2000 L product and a response is expected on or before February 28, 2009.