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Genzyme Myozyme BLA

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

Genzyme expects a priority review for its Myozyme (alglucosidase alfa) BLA for treatment of Pompe disease. The company submitted the application July 28, setting a priority review action date at Jan. 28. Pompe disease, which the firm estimates affects fewer than 10,000 people worldwide, is part of the same family of lysosomal storage disorders as mucopolysaccharidosis, Fabry and Gaucher disease...

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