Genzyme Corporation (Nasdaq: GENZ) announced today that it has received a complete response letter from the FDA regarding its application to market Lumizyme™ (alglucosidase alfa) for the treatment of Pompe disease. In its letter, the agency outlines the remaining items that need to be addressed before the application can be approved. Lumizyme is produced at the 2000 liter (L) bioreactor scale at Genzyme’s Allston Landing facility.
Specifically, Genzyme and the FDA must finalize agreement on the design of a post-approval verification study to demonstrate the clinical benefit of Lumizyme, as required under the Accelerated Approval process. In addition, Genzyme and the agency need to finalize the Risk Evaluation and Mitigation Strategy (REMS) for the product. Genzyme and the agency have been working closely and making progress toward these goals but were not able to reach them by the PDUFA date.
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