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US panel to review Genzyme's Myozyme for late-onset Pompe disease

This article was originally published in Scrip

Executive Summary

The US FDA's endocrinologic and metabolic drugs advisory panel will meet on October 21st to discuss Genzyme's BLA for Myozyme (alglucosidase alfa) for the treatment of late onset Pompe disease. The agency approved Myozyme in 2006 based on efficacy data in patients with infantile-onset disease, and Genzyme conducted a study in late-onset patients as a postmarketing commitment. The BLA was filed in May and is undergoing a priority review, with a November 29th user fee date. It seeks FDA approval to manufacture the enzyme-replacement therapy in a 2,000 litre bioreactor. The agency previously approved production at a 160 litre bioreactor, but the agency has determined that Myozyme produced at the two scales should be classified as different products because of differences in the carbohydrate structures. Agency approval of the BLA would result in the availability of two commercial versions of Myozyme in the US produced at different scales.

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