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Genzyme’s Pompe Program Update

Posted on: December 01, 2002

An update by Genzyme regarding treatment for Pompe disease.

Q: What is the status of the Pompe program?

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.

Q: When will new clinical trials begin?

Enrollment is expected to begin by the end of this year in the first of several planned clinical trials evaluating the use of rhGAA to treat infantile-onset Pompe disease. Patients up to three years old with the infantile-onset form of the disease will be included in this study. A second trial is expected to begin in the second quarter of 2003 and will focus on patients less than six months old with the most severe infantile-onset form of Pompe disease, in which infants generally die before the age of one year. The studies—to be conducted at medical centers in the United States, Europe, and Asia once we receive the necessary regulatory and IRB approvals—will focus on the effect of treatment on patient survival, as well as other factors such as respiratory function, cardiac status, motor development, and safety.  Clinical studies for patients with late-onset Pompe disease are expected to begin once a sufficient supply of clinical trial material is available.

Q: Are you able to make product available for compassionate use?

Sufficient quantities of Genzyme rhGAA are currently available to conduct the two planned infantile studies.  In addition, patients now receiving either transgenic rhGAA or rhGAA licensed from Synpac (North Carolina) Inc. are scheduled to transition to Genzyme rhGAA next year.  Genzyme has held discussions with regulatory authorities regarding the current limitations on product supply and the urgent needs of Pompe patients.  Until adequate product inventory exists, priority must be given to maintaining patients who are currently on therapy and to conducting the clinical studies necessary to secure product registrations worldwide.  Only with this focus can treatment be made available as quickly as possible to patients who need it.

Q: How is product manufacturing going?

Genzyme has successfully initiated production of rhGAA in its development-and pilot-scale manufacturing facilities in Framingham, Massachusetts.  In addition, Genzyme is now working to complete the installation of commercial-scale bioreactors at its Allston Landing manufacturing facility in Boston to provide capacity for rhGAA production in the longer term.

Q: When do you expect to seek product registration?

Genzyme is continuing its discussions with regulatory authorities in Europe regarding the content and timing of a marketing authorization application. These discussions have not yet been held with regulatory authorities in the United States or other countries.

Q: Have there been program management changes?

A: Yes.  John Crowley, senior vice president, Genzyme Therapeutics, and general manager of the Pompe program, is leaving the company to spend more time with his family, particularly his children, both of whom suffer from Pompe disease.  Mr. Crowley came to Genzyme in 2001 following Genzyme’s acquisition of Novazyme Pharmaceuticals Inc., where he was president and chief executive officer.  He will continue to work with Genzyme as a consultant. Frank Ollington, Ph.D., senior vice president, Genzyme Therapeutics, has assumed management responsibility for the Pompe program. Frank has held a number of important leadership positions in his 14 years with Genzyme.  He directed the start-up of the Allston Landing manufacturing plant, served as president of the Genzyme Pharmaceuticals business unit and senior vice president of corporate operations, and chaired the steering committee overseeing the development of Aldurazyme® (laronidase), a product under development for the treatment of the genetic disorder MPS 1.

This document contains forward-looking statements that are subject to risks and uncertainties that could cause actual results to differ materially from those projected in these forward-looking statements including, among others, the risks and uncertainties described in reports filed by Genzyme Corporation with the Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended, including without limitation Exhibit 99.2 to Genzyme’s 2001 Annual Report on Form 10-K, as amended.  These statements speak only as of the date of this document.  Genzyme® is a registered trademark of Genzyme Corporation.  Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.  All rights reserved.

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