Genzyme's Myozyme receives positive recommendation from European regulatory committee

By Angela McDaniels

Seattle, Jan. 27 - Genzyme Corp. said the Committee for Human Medicinal Products of the European Medicines Agency unanimously recommended full approval of Myozyme (alglucosidase alfa) for the treatment of Pompe disease.

Marketing authorization by the European Commission is expected within two to three months.

Pompe is a debilitating, progressive and often fatal disease affecting fewer than 10,000 people worldwide. Upon approval, Myozyme will be the first treatment for Pompe disease and one of the first for an inherited muscle disorder, according to a company news release.

Myozyme replaces acid alpha-glucosidase, an enzyme deficient in people with Pompe disease. As a result of this deficiency, people with the disease experience an excessive accumulation of glycogen in cell lysosomes which leads to impaired muscle function.

The marketing application for Myozyme included clinical data primarily from studies involving patients with infantile-onset Pompe disease, wherein symptoms typically appear within a few months of birth and babies frequently display a markedly enlarged heart and die within the first year of life.

In the late-onset form, symptoms may become evident anytime during childhood, adolescence or adulthood, and patients experience significant debilitation and premature mortality due to progressive respiratory failure.

Genzyme said it recently began a clinical trial evaluating the safety and efficacy of Myozyme in patients with late-onset Pompe disease designed to provide additional support for Myozyme's use. Results are expected to be available next year and will be submitted to regulatory authorities.

The company manufactures Myozyme at two facilities in the United States. To meet anticipated demand for the product, the company said it expects to also produce Myozyme in the future at its new protein manufacturing facility in Geel, Belgium.

Genzyme is a biotechnology company based in Cambridge, Mass., that develops therapeutics for rare inherited disorders, kidney disease, orthopedics, cancer, transplant and immune diseases and diagnostic testing.

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