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Print Edition StoriesThursday, December 3, 2009
Genzyme opens access to former FDA-rejected Pompe disease drug
By Mass High Tech staff
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Genzyme Corp. has reopened enrollment in its Alglucosidase Alfa Temporary Access Program (ATAP) for adults suffering from Pompe disease to obtain access to the company’s Lumizyme drug treatment prior to its approval for commercialization. Lumizyme has been rejected twice by the U.S. Food and Drug Administration, which pointed out Genzyme’s “deficiencies related to the Allston Landing manufacturing plant.”
As part of the reopening of enrollment, the FDA and Genzyme have agreed on a path to approve Lumizyme, the company reported in a press release. The path includes manufacturing the drug at a larger 4000 L bioreactor scale in the company’s Geel, Belgium-based plant. Genzyme officials said they plan to resubmit its Lumizyme application to the FDA this month.
Genzyme markets its Lumizyme treatment outside the U.S. as Myozyme, which is approved for commercialization in treating Pompe disease in more than 40 countries.
Genzyme’s Allston manufacturing plant was forced to shut down in the summer due to the discovery of a virus.
The FDA’s initial rejection of Lumizyme came before the Allston plant shut down and noted the need for an agreed-upon post-approval verification study and a finalized Risk Evaluation and Mitigation Strategy (REMS). The rejection notice included concerns of the Allston plant’s process controls, production equipment maintenance and microbiological monitoring and controls. Genzyme said it believes the other elements of the March review, such as the REMS, the product label, and post-marketing requirements, “have been satisfactorily addressed.”
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