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FDA approves new treatment for late-onset Pompe’s disease

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The FDA announced approval of aglucosidase alfa for patients aged 8 years and older with late-onset Pompe’s disease.

Aglucosidase alfa (Lumizyme, Genzyme) works by replacing deficient alpha-glucosidase, thereby reducing the accumulated glycogen in the heart and skeletal muscle cells.

“Pompe’s disease is a devastating condition without the appropriate treatment,” Julie Beitz, MD, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research, said in a press release. “The approval of Lumizyme will provide an important treatment for patients diagnosed later in life with Pompe’s disease.”

The drug is being approved with a risk evaluation and mitigation strategy. It will be available only through a restricted distribution system called the Lumizyme ACE (Aglucosidase Alfa Control and Education) Program to ensure that it is used by the correct patient group.

Aglucosidase alfa will also carry a boxed warning because of the risk for anaphylaxis, severe allergic reactions and immune-mediated reactions.

The safety and efficacy of aglucosidase alfa are based on a clinical study of 90 patients aged 10 to 70 years with late-onset Pompe’s disease. The most commonly reported adverse events were infusion-related reactions and included severe allergic reactions, hives, diarrhea, vomiting, shortness of breath, itchy skin, skin rash, neck pain, partial hearing loss, flushing, pain in extremities and chest discomfort. However, the safety and efficacy of aglucosidase alfa in patients with infantile-onset Pompe’s disease or in patients aged 8 years or younger with late-onset disease have not been evaluated.

Some adult patients in the United States received aglucosidase alfa under a temporary access program. Approval of this drug will ensure that treatment is available for all U.S. adults with Pompe’s disease in need of treatment, according to the FDA.

The only other currently available treatment for Pompe’s disease is Myozyme, also manufactured by Genzyme. This drug has been in short supply because of limited manufacturing capacity, the agency said.

It is estimated that Pompe’s disease occurs in one in every 40,000 to 300,000 births.

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