Drug Approved for Pompe Disease After Priority Review

Rare Pompe disease usually fatal in children

MONDAY, May 1 (HealthDay News) -- The U.S. Food and Drug Administration (FDA) has approved the first treatment for Pompe disease, which affects one in 40,000 to 300,000 children, and results in a deficiency in the enzyme acid alpha-glucosidase. Myozyme (alglucosidase alfa) has been approved for intravenous administration based on clinical trials in 39 patients aged 1 month to 3.5 years at the time of the first infusion.

Trials showed that infants treated with the drug were more likely to survive without invasive ventilation support compared with untreated patients.

Heart and lung failure, as well as allergic shock, are the most severe side effects of Myozyme, although more common side effects were pneumonia, respiratory failure and distress, infections and fever. The FDA notes that Myozyme will include a boxed warning about the possibility of life-threatening allergic reactions.

Myozyme has been given FDA Orphan Drug designation and was approved under a priority review. Myozyme is manufactured by Genzyme Corporation in Cambridge, Mass.

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