Genzyme gets European Commission approval for Myozyme to treat Pompe disease

By Elaine Rigoli

Tampa, Fla., April 3 - Genzyme Corp. has received European Union marketing authorization for Myozyme (alglucosidase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Pompe disease, a debilitating, progressive and often fatal disorder affecting fewer than 10,000 people worldwide.

The product is the first treatment approved for Pompe disease and one of the first for an inherited muscle disorder, according to a news release.

Pompe disease manifests as a broad spectrum of clinical symptoms. All patients typically experience progressive muscle weakness and breathing difficulty, but the rate of disease progression can vary widely depending on the age of onset and the extent of organ involvement, the release said.

"This is an extraordinary moment for Pompe patients and their families. The effort to develop Myozyme has required the enormous commitment of many people throughout Genzyme and across the Pompe community, who have worked with a great sense of urgency and have overcome tremendous challenges. Our focus now is to ensure that Myozyme is available to all patients who need treatment," said Henri A. Termeer, chairman and chief executive officer, in a statement.

Myozyme has received orphan medicinal product designation in Europe.

Genzyme said it will introduce Myozyme in Europe on a country-by-country basis, as pricing and reimbursement approvals are obtained.

Genzyme manufactures Myozyme at two facilities in the United States. To ensure that it is able to meet the anticipated demand for the product in Europe and throughout the world, the company said it expects to produce Myozyme at its new protein manufacturing facility in Geel, Belgium, and its new fill/finish facility in Waterford, Ireland.

Genzyme products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune diseases and diagnostic testing. The company is based in Cambridge, Mass.

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