Genzyme receives FDA complete response letter for its Lumizyme marketing application

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 stated that satisfactory resolution of deficiencies related to the Allston Landing manufacturing plant are required before the Lumizyme application can be approved. Genzyme believes the other elements of the Lumizyme review, such as the Risk Evaluation and Mitigation Strategy (REMS), the product label, and post-marketing requirements, have been satisfactorily addressed.

The FDA completed its five-week inspection of the Allston plant on Friday, and provided Genzyme with a Form-483 outlining remaining deficiencies, which were mainly related to the fill/finish capabilities at the facility. The FDA has indicated a willingness to work with Genzyme to resolve these issues and to ensure continued product supply. Genzyme’s plan to address these deficiencies includes establishing additional internal controls and updating fill/finish capabilities in Allston, transferring additional filling activities to existing Genzyme contract manufacturers, and utilizing excess capacity at Genzyme’s manufacturing facility in Waterford, Ireland. The state-of-the-art Waterford facility is undergoing a major expansion, with engineering runs scheduled to begin in early next year. This expansion will increase Genzyme’s internal filling capacity by four fold. Cerezyme® (imiglucerase for injection) 400-unit vials, which supply over 80 percent of patients worldwide, will be filled exclusively in Waterford moving forward.

Alglucosidase alfa 2000 L scale (Lumizyme) is no longer produced at the Allston facility and Genzyme is transitioning all patients worldwide to the product produced at a larger scale in the company’s facility in Geel, Belgium. Production of alglucosidase alfa at the larger 4000 liter bioreactor scale is required to fulfill global demand. Genzyme will be requesting a meeting with FDA to discuss the most expeditious path forward toward U.S. approval of the product produced at this larger scale in Belgium.

“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 Genzyme Chairman and Chief Executive Officer Henri Termeer.

Supply Update

Genzyme has successfully processed material to final product stage for both Cerezyme and Fabrazyme® (agalsidase beta). A second set of Fabrazyme runs has been initiated. Genzyme continues to anticipate making a Cerezyme shipment later this month that will enable patients currently receiving product to continue to be treated. Cerezyme shipments in December will allow all patients access to treatment at the end of the month. Genzyme plans to communicate more specific details around the dose and timing of Cerezyme re-supply in the next ten days. Genzyme continues to anticipate making a Fabrazyme shipment at the end of December, which will allow patients to continue therapy at current levels. The company is currently evaluating the impact of changes to its fill/finish operations on Fabrazyme, and Genzyme expects to communicate timelines and further details regarding Fabrazyme resupply by early December.