FDA To Fast-Track Genzyme Application for Myozyme

The FDA plans an Accelerated Approval for Genzyme’s Myozyme drug, produced at the 2000 L bioreactor scale for the treatment of Late Onset Pompe disease. According to a Genzyme statement, the two parties need to agree on the design of a post-approval verification study and the FDA must complete its review of the Risk Evaluation and Mitigation Strategy (REMS) for the product. Genzyme submitted the REMS earlier this month. The FDA has classified this submission as a major amendment to the BLA for Myozyme produced at the 2000 L scale, and has extended the PDUFA date by 90 days to February 28, 2009. Genzyme will be required to submit the final protocol for the verification study after approval.

Genzyme currently has U.S. approval to sell Myozyme produced at the 160 L bioreactor scale, and the company has been seeking clearance from the FDA for 2000 L-scale production. Genzyme submitted a separate BLA for the product using the 2000 L manufacturing process on May 30, following a determination by the FDA that Myozyme produced at the 160 L and 2000 L scales should be considered as two separate products because of comparability differences.

“We’re very pleased to have additional clarity from the FDA on what it will take for 2000 L-scale product approval,” said Genzyme’s senior vice president of Global Market Access, Alison Lawton. “The ongoing communication with the FDA has been positive, and the agency has done a substantial amount of work in a short time to facilitate approval of this product for patients.”

Myozyme is a treatment for Pompe disease, a progressive, debilitating and life-threatening inherited disorder affecting approximately 2,000 people in the U.S. As part of the accelerated approval procedure, a verification study is required to demonstrate clinical benefit of the 2000 L-scale product during the post-marketing period.

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