FDA Grants Marketing Approval For Genzyme's Lumizyme
The company claims that Lumizyme is the first treatment approved in the US specifically to treat patients with late-onset Pompe disease.
According to the company, Lumizyme is a lysosomal glycogen-specific enzyme indicated for patients eight 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 trials in infantile-onset patients, or in late (non-infantile) onset patients less than eight years of age.
Genzyme has worked closely with patients and physicians in the US Pompe community during the preapproval period to assure that the most severely affected late-onset patients could access therapy in advance of Lumizyme approval.
Genzyme will now work closely with the treating centers and prescribers to insure that patients in the ATAP program can continue to access therapy during the transition to commercial supply. Genzyme will also begin working with US healthcare professionals to help adult patients who have been waiting to access treatment.
In effort to preserve 160-L scale product for infantile-onset patients, Genzyme will begin to transition eligible patients who are receiving Myozyme onto Lumizyme.
Henri Termeer, chairman and CEO of Genzyme, said: “This is an important day for the Pompe community, especially for those patients with late-onset Pompe disease in the United States who are awaiting treatment for this devastating disease. We are grateful to the FDA for their efforts to approve Lumizyme ahead of its scheduled PDUFA date.”
Will this approval help Genzyme to provide better patient care?
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