Genzyme Pompe Program Update—November 2008
Posted on: November 17, 2008
The US Food and Drug Administration (FDA) has informed Genzyme that the FDA plans an accelerated approval for alglucosidase alfa produced at the 2000 L bioreactor scale for the treatment of late onset Pompe disease. Prior to approval, the company and the agency first 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), which Genzyme submitted earlier this month. This REMS program is intended to ensure that distribution of the 2000 L product is prescribed and administered to the intended patient population. The FDA has classified this submission as a major amendment to the Biologics License Application (BLA) for alglucosidase alfa produced at the 2000 L scale. Therefore, to provide time to agree on the design of a post-approval verification study and complete the review of the REMS submission, the FDA has changed the Prescription Drug User Fee Action (PDUFA) date to February 28, 2009. Genzyme will be required to submit the final protocol for the verification study after approval.