Pompe Program Update

Category: Archive
December 16, 2002

Genzyme’s Pompe Program Update Q&A, December 2002

This Question and Answer session is derived from December’s IPA/Genzyme teleconference

Q: What is the status of the Pompe program?

A: Genzyme has made strong progress within its Pompe disease program since its decision earlier this year to move forward with an internally developed product candidate. In November, we filed an IND with the FDA for the first of several planned clinical studies, marking a significant milestone in our Pompe program. We share with patients and their families a tremendous sense of urgency to begin clinical studies of Genzyme’s recombinant human acid alpha-Glucosidase (rhGAA), so that we can move toward the goal of making a treatment broadly available. Our discussions with regulatory authorities to date have been positive, and we are optimistic that they will support us in moving this process forward expeditiously. The Pompe program is currently Genzyme’s largest research and development initiative.

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