Genetics

27 May 2010

Lumizyme Approved for Non-Infantile Pompe Disease

A rare genetic disorder

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TUESDAY, May 25 (HealthDay News) -- Lumizyme (alglucosidase alfa) has been approved by the U.S. Food and Drug Administration to treat late-onset Pompe disease, a genetic disorder that often leads to fatal respiratory failure.

Pompe, affecting one in every 40,000 to 300,000 births, is caused by a gene mutation that prevents the body from making enough of an essential enzyme. This leads to heart and skeletal muscle weakness that commonly progresses to respiratory failure.

Lumizyme is thought to replace the deficient enzyme, acid alpha-glucosidase (GAA), the FDA said in a news release. The approval sanctions the drug for people aged 8 and older with non-infantile (late-onset) Pompe disease.

The drug will carry a boxed label warning of potential side effects including anaphylaxis and severe allergic reactions, the FDA said. Other commonly reported side effects include hives, diarrhea, vomiting, shortness of breath, itchy skin or rash, neck pain, partial hearing loss, flushing, extremity pain and chest discomfort.

Lumizyme will be made available through a restricted distribution system "to ensure that it is used by the correct patient group," the agency said.

The drug is produced by Genzyme, based in Cambridge, Mass.

More information

The U.S. National Institute of Neurological Disorders and Stroke has more about Pompe Disease.

 

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