CAMBRIDGE, Mass – Genzyme General (Nasdaq: GENZ), a division of Genzyme Corp., announced today that it has begun enrolment in a clinical trial evaluating the use of its Myozyme™ recombinant human acid alpha -Glucosidase enzyme as a potential treatment for Pompe disease.
The trial is the first of two studies Genzyme expects to conduct this year involving patients with the infantile-onset form of Pompe disease, in which symptoms manifest themselves during the first year of life. The trial (known as study 1702) will include up to 16 children between the ages of six months and three years. It is being conducted at medical centers in the United States and Europe.
A second trial (known as study 1602) is scheduled to begin this spring, and will include up to 16 infants younger than six months of age at the time of their first infusion. Genzyme expects to complete enrolment in both studies this year. The studies will focus on the effect of Myozyme on patient survival, as well as other factors such as respiratory function, cardiac status, motor development, and safety. Genzyme expects to pursue product registrations globally based on results from these trials and previous studies of enzyme replacement therapy for Pompe disease.
“We are moving forward with sense of urgency to complete these studies,” said Henri A. Termeer, chairman and chief executive officer of Genzyme Corp. “This is a devastating disease, and we are committed to doing all that is necessary to bring forward a safe and effective treatment for patients.”
About Pompe disease
Pompe disease is a rare and fatal genetic disorder caused by a deficiency of the lysosomal enzyme acid alpha- Glucosidase, which is responsible for breaking down glycogen within cells. Pompe disease shares a common element of muscle wasting with more than 40 different muscle diseases, often called muscular dystrophies. There is currently no treatment available for this disease, which affects several thousand people worldwide. Pompe disease ranges from a rapidly fatal infantile-onset form with severe cardiac involvement to a more slowly progressive adult-onset form.
Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General has five therapeutic products on the market and a strong pipeline of therapeutic products in development focused on the treatment of genetic diseases and other chronic debilitating disorders with well-defined patient populations. Genzyme General is a division of Genzyme Corp.
This press release contains forward-looking statements, including statements about: planned clinical trials for a potential therapy for Pompe disease, including the anticipated timing and design of trials; plans regarding submissions to regulatory authorities; and manufacturing plans and expectations. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected in these forward-looking statements. These risks and uncertainties include, among others: the receipt of IRB approvals for clinical trials and the timing thereof; enrolment rates for clinical trials; the efficacy and safety of Genzyme rhGAA in humans and the actual timing and final design of clinical trials; the results of pre-clinical and clinical studies; the actual timing and content of submissions to and decisions made by regulatory authorities concerning the Pompe product candidate and facilities, including the nature and extent of data required for marketing applications; the results and timing of qualifying runs at facilities; the ability to manufacture sufficient quantities of product for development activities (including without limitation extension studies and expanded access arrangements) and to do so in a timely and cost-efficient manner; and the risks and uncertainties described in reports filed by Genzyme 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. Genzyme General Division common stock is a series of common stock of Genzyme Corporation. Therefore, holders of Genzyme General Division common stock are subject to all of the risks and uncertainties described in the those reports. We caution investors not to place undue reliance on the forward-looking statements contained in this press release. These statements speak only as of the date of this press release, and we undertake no obligation to update or revise the statements. Genzyme® is a registered trademark of Genzyme Corporation. All rights reserved.