Therapy — It’s Here, and Free
Letters to the Editor
15 August 2007
The Wall Street Journal
The Wall Street Journal – Print and Online
Genzyme is working closely and constructively with the Food and Drug Administration to obtain
approval of the large-scale manufacturing process to supply Myozyme, our therapy for Pompe
disease, to the U.S. market (“Genzyme’s Hit Turns Headache,” Heard on the Street, Aug. 9). As
typically happens in such reviews, the agency has asked for additional information, which we will
supply in the coming months. We anticipate approval of our large-scale manufacturing process in
the first half of 2008.
Because Pompe is a life-threatening disease, Genzyme has been working diligently with patients,
physicians and the FDA to manage our existing U.S. supply during this review period and to help
ensure that we are able to treat the most severe, life-threatening cases.
In collaboration with the FDA, we have implemented a program to provide therapy to these
patients free of charge. This program employs Myozyme produced from the large-scale
manufacturing process that is currently approved in 33 countries throughout the world, but not yet
in the U.S. We are confident based on all available data, including the clinical experience of more
than 500 patients currently benefiting from Myozyme produced at this larger scale, that there is
no difference in the safety or effectiveness of the two sources.
We will continue to work closely with the FDA, patients and physicians to ensure patients have
access to the medicine they need.
David Meeker, M.D.
Lysosomal Storage
Disorder Therapeutics
Genzyme Corp.
Cambridge, Mass.