Genzyme Pompe Program Update—April 2008
Posted on: April 21, 2008
We would like to take this opportunity to provide an important update related to Myozyme (alglucosidase alfa) manufacturing in the United States. The FDA has informed Genzyme of its decision on the application for the larger scale manufacturing process to commercially supply Myozyme in the US. The FDA has decided that Myozyme produced at the smaller bioreactor scale (160 L) and at the larger scale (2000 L) should be classified as two different products based on a comparison of biochemical characteristics of the product produced at the two scales. We at Genzyme are disappointed in this decision. Genzyme believes that both products are clinically effective and safe. The product from the smaller scale was used in the pivotal trials supporting FDA approval of Myozyme. The product from the larger scale is approved in 41 countries and is currently used to treat approximately 900 patients of all ages throughout the world including the 138 adults in the US who are currently accessing Myozyme through the Myozyme Temporary Access Program (MTAP).