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 ... More), produced at the 4000 liter (L) bioreactor scale at its manufacturing facility in Geel, Belgium. LumizymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More 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. More.
“This is an important day for the Pompe community, especially for those patients with late-onset Pompe diseaseA form of Pompe disease that begins after infancy and usually progresses more slowly. More 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 LumizymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More ahead of its scheduled PDUFA date.”
LumizymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) is a lysosomal glycogen-specific enzymeA protein that helps the body carry out chemical reactions. More indicated for patients 8 years and older with late (non-infantile) onset Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More (GAA deficiency) who do not have evidence of cardiac hypertrophy. The safety and efficacy of LumizymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More have not been evaluated in controlled clinical trialsA research study that tests new treatments or approaches in people. More in infantile-onset patients, or in late (non-infantile) onset patients less than 8 years of age.
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