Genzyme Gets FDA Nod for Pompe Drug
Cambridge, MA-based Genzyme (NASDAQ:GENZ) has won FDA approval to sell its Pompe disease drug alglucosidase alpha (Lumizyme) made in 4,000-liter batches at its plant in Geel, Belgium, the company reported this morning. The FDA approved the drug for treating patients who are at least 8 years old with Pompe, a rare genetic disease that robs people of their ability to make an enzyme that breaks down sugars that build up and enlarge heart and muscle tissues. Genzyme’s drug replaces the lacking enzyme. The disease, which can be fatal, causes symptoms such as lung infections and respiratory failure, according to the National Institutes of Health.