The Food and Drug Administration officials have declined the approval of Genzyme Corp.’s new Lumizyme treatment for Pompe disease on the grounds that there are still some issues that need to be addressed by the company before the application can be approved.

 

More exactly, Genzyme and the FDA must finalize agreement on the design of a post-approval verification study to prove Lumizyme’s clinical benefit, as required under the Accelerated Approval process. In addition to that, the company and the agency need to finalize the Risk Evaluation and Mitigation Strategy for the drug.

 

The FDA went even further and issued a warning letter to Genzyme after discovering problems during inspections last fall at the bigger Boston facility where the company hopes to produce Lumizyme. The FDA letter specifically noted deficiencies related to microbiological monitoring and controls, equipment maintenance and process control. These issues must be resolved before the FDA will approve the Boston facility, the company said.

 

“We are obviously surprised and disappointed by this further delay. We are confident we will be able to resolve all remaining issues with the FDA within three to six months,” said Henri Terneer, Genzyme's chief executive.

 

Pompe disease is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body’s cells. The accumulation of glycogen in certain tissues, especially muscles, impairs their ability to function normally. The disease affects about 1 in 40,000 people.