FDA Shoots Down Genzyme’s Latest Bid for Pompe Drug Approval

11/16/09

Even more bad news from Genzyme (NASDAQ:GENZ). Following the news on Friday about new contamination found in treatments made at the Cambridge, MA-based biotech firm’s Allston, MA, plant, the company says today the FDA informed it that the agency won’t approve its application to market Pompe disease drug alglucosidase alfa (Lumizyme) made in large-scale batches until it addresses deficiencies at the facility.

Genzyme, which already has approval to make the drug in smaller bioreactors, said that it plans to request a meeting with the FDA to discuss what needs to be done to garner approval of the product made in larger containers. Meantime, the firm is making the drug in smaller batches under the name Myozyme. Genzyme plans to hold a conference call at 12 pm ET to discuss the FDA’s response.

The FDA is forcing Genzyme to get an additional approval of alglucosidase alfa—an enzyme replacement therapy for patients with rare lysosomal storage disorder—made in larger bioreactors because slight changes in manufacturing process such as the batch size can have an impact on the finished product. Back in April 2006, the treatment was the first of its kind approved for Pompe disease in the U.S., and it’s been one of Genzyme’s fastest-growing sellers. The company says that it needs approval of the treatment made in 4,000-liter batches at a factory in Belgium to meet global demand for the treatment. And this isn’t the only Genzyme drug in short supply. The company has experienced shortages of its top sellers, Cerezyme and Fabrazyme, after a viral contamination struck its Allston factory in June.

“We have made significant progress in bringing the Allston plant back in operation, and we will continue to work closely with the FDA to resolve these issues,” said Henri Termeer, Genzyme’s chairman and CEO, in a statement.

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