New Treatment for Pompe Disease Approved

WASHINGTON, May 25 (UPI) — The U.S. Food and Drug Administration announced approval Tuesday of a new treatment for some patients with late-onset (non-infantile) Pompe disease.

The FDA said the rare genetic disorder produces heart and skeletal muscle weakness that progresses to respiratory failure.


The federal agency said it approved Lumizyme (alglucosidase alfa) for patients ages 8 years and older with the disease in which a gene mutation prevents the body from making an enzyme, or making enough of the enzyme (acid alpha-glucosidase) for proper muscle functioning.

Lumizyme is believed to work by replacing the deficient enzyme, thereby reducing the accumulated glycogen in heart and skeletal muscle cells, officials said.

The FDA said Lumizyme will carry a Boxed Warning because of the risk of severe allergic and immune-mediated reactions.

Currently, the only other treatment for Pompe disease available in the United States is Myozyme, which has been in short supply due to limited manufacturing capacity.

Both Myozyme and Lumizyme are marketed by Genzyme of Cambridge, Mass.

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