News & Information:

Health Canada approves first life-saving enzyme replacement therapy for Fabry Disease

Genzyme's ^(Pr)Fabrazyme^® Now Available for Canadians with Rare, Fatal Disease

Date: February 10, 2004

Up until now, people with Fabry disease have had one certainty... death in their forties or fifties, preceded by years of prolonged suffering. With the approval of ^(Pr)Fabrazyme^® (agalsidase beta), Canada's first enzyme replacement therapy and treatment option for Fabry disease, Genzyme Canada, a wholly-owned subsidiary of Genzyme Corp., gives new hope to Fabry patients in Canada.

"This is a great day for Canadians with Fabry disease," said Richard Corkum, founder of the Fabry Society of Canada. "I am overjoyed that Fabrazyme is now available in Canada and that people across the country will have access to life-saving enzyme replacement therapy."

Fabry disease is a rare, life-threatening, inherited metabolic disorder caused by an abnormal or faulty gene in the body. In Canada, it is estimated that more than 100 people have been diagnosed with Fabry disease. While predominantly affecting males, (inherited through the Fabry gene located on the X chromosome), females can also suffer the serious consequences of Fabry disease.

"Approval of Fabrazyme marks a tremendous milestone for the many diagnosed and non-diagnosed Fabry sufferers in Canada who will finally have treatment for this debilitating disease," said Dr. Wyatt, geneticist and one of the principal investigators in Canada's review of the effectiveness of Fabrazyme. "Now we will not just manage Fabry symptoms of pain and end-stage complications of kidney failure, cardiac disease and strokes. With Fabrazyme, we can actually treat the disease itself and help alleviate the devastating impact Fabry disease has on patients and their families. It is marvelous that Canada has now joined the ranks of numerous other countries which have confirmed the value of this therapy in improving patients' lives."

Due to a deficiency in an important enzyme, alpha-galactosidase A or alpha-GAL, Fabry disease causes certain fats to accumulate in the blood vessels over many years. The ensuing buildup of fatty substance globotriaosylceramid (GL-3) compromises the cells of various tissues, including organs such as the kidneys and heart, eventually leading to complete organ collapse. Fabry patients must often cope with significant pain and disability and typically have shortened life spans, with the average life expectancy in the mid forties.

"I was diagnosed with Fabry disease in January 2001, after my kidneys failed," said Adrian (Ed) Koning, a Fabry patient from Edmonton, Alberta. "Fabrazyme has definitely had a positive impact on my life. It is a necessary, life-saving drug for Fabry patients and I'm happy to finally see it approved in Canada."

Fabrazyme, an enzyme replacement therapy, actually substitutes the missing or malfunctioning enzyme in Fabry patients. Administered by an intravenous infusion of a genetically engineered DNA form of alpha-GAL, Fabrazyme initiates the breakdown of GL-3 accumulation in the cells allowing the body to rid itself of this excess buildup of harmful fatty substances through the blood stream.

"Fabrazyme has been proven to be highly effective in removing the accumulated GL-3 in Fabry disease," said Dr. Wyatt.

Guidelines for the management of Fabry patients have recently been developed by a group of Canadian experts in Fabry Disease. The group, chaired by Dr. Lorne Clarke, Medical Director Provincial Medical Genetics Program, BC, has submitted the document for publication in the Canadian Medical Association Journal where it is pending review.

Studied in the world's largest (58 patients), placebo controlled, randomized, multi-centred clinical trial in the history of treatment of this kind of genetic disease, evidence has shown that Fabrazyme reduces GL-3 accumulations in the kidney and certain other cell types⁽¹⁾. In fact, a study published in The New England Journal of Medicine concludes that Fabrazyme cleared (reduced to normal or near-normal levels) GL-3 deposits from the kidneys, heart and skin in patients with Fabry disease, reversing the origin and development of the main symptoms of the disease^(2). Patients treated with Fabrazyme experienced a sustained reduction in pain, a reduction in pain medication, an increase in episode free days, an increase in symptom free days, and an increase in quality of life⁽³⁾.

Health Canada has granted Genzyme a Notice of Compliance (NOC) for Fabrazyme without conditions.

"Genzyme has been researching and refining this product for years," said Paul Drohan, vice-president and general manager of Genzyme Canada Inc. "and we are extremely proud to be able to spearhead this revolutionary treatment for Canadians suffering from Fabry disease. Now, with the availability of Fabrazyme, Fabry patients can live longer, healthier lives."

Fabry disease belongs to a family of approximately 40 rare, inherited diseases known as lysosomal storage disorders. Genzyme is a leader in the development of treatments for these "ultra-orphan" diseases, all of which affect fewer than 10,000 people worldwide. In 1991, Genzyme introduced the first treatment for Gaucher disease, an enzyme replacement therapy that is now marketed in 60 countries.

About Genzyme

Genzyme Corporation is a biotechnology and health care products company that develops innovative products and services for major unmet medical needs. Genzyme Canada Inc., located in Mississauga, Ontario, is the Canadian affiliate of Genzyme Corp., headquartered in Cambridge, Massachusetts.

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(1) N Engl J Med 2001;345:9-16, July 5, 2001 (2) N Engl J Med 2001;345:9-16, July 5, 2001 (3) N Engl J Med 2001;345:9-16, July 5, 2001

For further information: Heather Grant, Courtney Rainey Group, T: (416) 815-9100 x230, heather@courtneyraineygroup.com; Sharon Rainey, Courtney Rainey Group, T: (416) 815-9100 x229, Sharon@courtneyraineygroup.com; Gabrielle Collu, Courtney Rainey Group, T: (514) 844-7338, collug@videotron.ca

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