Early treatment with Fabrazyme® significantly slows progression of Fabry

Brussels, Belgium – Treatment with Genzyme’s enzyme replacement therapy Fabrazyme (agalsidase beta) significantly decreases the progression of Fabry disease, as demonstrated by a clinical study published in a recent issue of the Annals of Internal Medicine. [1] The largest, longest placebo-controlled study ever conducted in Fabry disease measured the effectiveness of Fabrazyme in reducing the risk of major clinical events such as renal, cardiac and cerebrovascular events in patients with advanced Fabry disease.

Patients receiving the recommended 1mg/kg dosage of Fabrazyme saw a reduction of over 60 percent in the risks of major adverse events linked to the progression of Fabry disease compared to the control group. In addition, the study demonstrated the advantage of early treatment with Fabrazyme, as patients with no signs of severe kidney damage (nephropathy) experienced a reduced risk of major clinical events of over 80 percent, compared to a 15 percent reduction in patients with advanced nephropathy. [2] In this study Fabrazyme was generally well tolerated and no new (significant) safety concerns were identified.

“This is concrete evidence of the significant benefits Fabrazyme treatment brings to Fabry patients,” says Khazal Paradis, Senior VP Clinical Research Europe for Genzyme. “Because renal, cardiac, and cerebrovascular events are the predominant driver of morbidity and mortality for patients with Fabry disease, this treatment has the potential to greatly improve their quality and length of life.”

Fabry is one of a group of rare diseases called lysosomal storage disorders. The primary pathology of the disease is the accumulation of certain lipids in various cell types. It is a severe, progressive and multi-systemic disease which initially manifests in neurological symptoms, leading to high mortality and morbidity after the age of 30 due to renal, cardiac, and cerebrovascular events. [3]

One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 9,000 employees in locations spanning the globe. Genzyme has been selected by FORTUNE as one of the “100 Best Companies to Work for” in the United States.

With many established products and services helping patients in more than 80 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune diseases, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as heart disease and other areas of unmet medical need.

Genzyme® is a registered trademark of Genzyme Corporation. All rights reserved.

References

1. Banikazemi M, Bultas J, Waldek S, Wilcox WR et al. Ann. Intern Med 2007;146:77-86: Agalsidase-Beta Therapy for Advanced Fabry Disease - A Randomized Trial;

2. In patients with a baseline estimated glomerular filtration rate (eGFR) over 55 ml/min/1.73m² ; an intention-to-treat, proteinuria adjusted analysis showed a risk reduction of 81% (hazard ratio 0.19 [p=0.025], versus 15% in the group with an eGFR below 55 ml/min/1.73m².

3. Eng CM, Germain DP, Banikazemi M, Warnock DG, Wanner C, Hopkin RJ, et al. Fabry disease: guidelines for the evaluation and management of multi-organ system involvement. Genet Med. 2006;8(9): 539-48.