Genzyme Corp. has received marketing authorization for Myozyme (alglucosidase alfa) in the European Union.

Myozyme has been approved 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 ever approved for Pompe disease and one of the first for an inherited muscle disorder, according to a company release.

Henri A. Termeer, chairman and chief executive officer of Genzyme Corp said, "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."

Myozyme has received orphan medicinal product designation in Europe. The orphan medicinal products regulation is designed to encourage the development of treatments for rare disorders such as Pompe disease, for which no therapies have existed previously. Genzyme 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 expects to also produce Myozyme in the future at its new protein manufacturing facility in Geel, Belgium, and its new fill/finish facility in Waterford, Ireland, stated the release.

Pompe disease, also known as Acid Maltase Deficiency or Glycogen Storage Disease Type II, is one of more than 40 genetic diseases called lysosomal storage disorders, which are caused by a deficiency or malfunction of specific enzymes found in cell lysosomes.