(RTTNews) - Genzyme Corp. (GENZ: News ) announced that it has received a complete response letter from the FDA regarding its application to market Lumizyme, or 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 or 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. 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.

The company said that 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.

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by RTT Staff Writer

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