FDA approved Mesozyme for Pompe disease
The Food and Drug Administration today approved a biologics license application (BLA) for Myozyme (alglucosidase alfa, rhGAA), the first treatment for patients with Pompe disease,
Myozyme had been granted FDA Orphan Drug designation and was approved under a priority review.
The FDA approved Myozyme for administration by intravenous infusion of solution into a vein.
The safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion.
The Myozyme label includes the following indication: "Myozyme (alglucosidase alfa) is indicated for use in patients with Pompe disease (GAA deficiency). Myozyme has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control, whereas use of Myozyme in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy
Myozyme is manufactured by Genzyme Corp. in Cambridge, Mass.
More details about myozyme from Genezyme
Source
Genezyme
FDA
Myozyme had been granted FDA Orphan Drug designation and was approved under a priority review.
The FDA approved Myozyme for administration by intravenous infusion of solution into a vein.
The safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion.
The Myozyme label includes the following indication: "Myozyme (alglucosidase alfa) is indicated for use in patients with Pompe disease (GAA deficiency). Myozyme has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control, whereas use of Myozyme in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy
Myozyme is manufactured by Genzyme Corp. in Cambridge, Mass.
More details about myozyme from Genezyme
Source
Genezyme
FDA
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