FDA Approves Lumizyme to Treat Rare Pompe Disease

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Lumizyme (alglucosidase alfa), produced by Massachusetts based Genzyme, has been approved by the US Food and Drug Administration (FDA) to treat late-onset Pompe disease. The drug will be made available through a restricted distribution system “to ensure that is used by the correct patient group,” the agency said.

Pompe Disease is a rare, genetic disease that is often fatal. It is caused by a mutation in a gene that makes alpha-glucosidase (GAA), an enzyme that breaks down glycogen, a stored form of sugar used for energy. When this enzyme is reduced or eliminated, excessive amounts of glycogen are stored in the body cells, particularly those of the heart and skeletal muscles. The condition is estimated to occur in 1 in every 40,000 births.

Read: "Extraordinary Measures" Brings Awareness to Rare Pompe Disease

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Late onset Pompe disease is the result of a partial deficiency of GAA, while infantile Pompe disease (or early onset) is a complete or near complete deficiency of the enzyme. Most babies with infantile Pompe disease die from cardiac or respiratory complications before their first birthday. The primary symptom of the late-onset form of the disease is muscle weakness that progresses to respiratory weakness and death from respiratory failure.

Lumizyme is approved for people aged 8 and older with late-onset (non-infantile) Pompe disease who do not have evidence of cardiac hypertrophy (enlarged heart). The drug is a form of another Pompe disease medication called Myozyme, approved for the infantile onset disease. Both medications are alpha-glucosidase enzyme replacements that, in clinical trials, reduces glycogen accumulation and improves muscle function, tone, and strength.

The drug is only available to patients enrolled in a restricted distribution program called the Lumizyme ACE program . Because it is a protein, the drug must be administered by infusion every two weeks. It will carry a boxed label warning, called a black box warning, of potential side effects that include anaphylaxis and severe allergic reactions. Other common side effects include hives, diarrhea, vomiting, shortness of breath, itchy skin or rash, neck pain, partial hearing loss, flushing, extremity pain, and chest discomfort.

For more information or to enroll in the Lumizyme ACE Program, call (800) 745-4447.

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