CAMBRIDGE, Mass. – Genzyme Corporation (Nasdaq: GENZ) announced today that the FDA has granted U.S. marketing approval for LumizymeTM (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme.), produced at the 4000 liter (L) bioreactor scale at its manufacturing facility in Geel, Belgium. Lumizyme is the first treatment approved in the United States specifically to treat patients with late-onset Pompe diseaseA form of Pompe disease that begins after infancy and usually progresses more slowly..
“This is an important day for the Pompe community, especially for those patients with late-onset Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. in the United States who are awaiting treatment for this devastating disease,” said Genzyme Chairman and Chief Executive Officer, Henri A. Termeer. “We are grateful to the FDA for their efforts to approve Lumizyme ahead of its scheduled PDUFA date.”
Lumizyme (alglucosidase alfa) is a lysosomal glycogen-specific enzymeA protein that helps the body carry out chemical reactions. indicated for patients 8 years and older with late (non-infantile) onset Pompe disease (GAA deficiency) who do not have evidence of cardiac hypertrophy. The safety and efficacy of Lumizyme have not been evaluated in controlled clinical trialsA research study that tests new treatments or approaches in people. in infantile-onset patients, or in late (non-infantile) onset patients less than 8 years of age.
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