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🏅 FDA Orphan Designation

Myozyme

Recombinant human acid alpha-glucosidase; alglucosidase alfa

Manufacturer: Genzyme Corporation

Indicated for:
Glycogen storage disease due to acid maltase deficiencyOrphanInfantile Refsum disease

FDA-Approved Indications (2)

Myozyme for use in patients with Pompe disease (GAA deficiency). Alglucosidase alfa has been shown to improve ventilator-free survival in patients with infantile onset Pompe disease as compared to an

Myozyme for use in patients with Pompe disease (GAA deficiency). Alglucosidase alfa has been shown to improve ventilator-free survival in patients with infantile onset Pompe disease as compared to an

Population: infantile onset

Indications & Usage

Myozyme for use in patients with Pompe disease (GAA deficiency). Alglucosidase alfa 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 Alphaglucosidase in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy. ||| Lumizyme 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 (alglucosidase alfa) have not been evaluated in controlled clinical trials in infantile-onset patients, or in late (non-infantile) onset patients less than 8 years of age ||| (Lumizyme) Patients with Pompe disease (acid alpha-glucosidase (GAA deficiency)).

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Myozyme
Genzyme Corporation
Myozyme
Genzyme Corporation

Where shown, WAC (Wholesale Acquisition Cost) is an estimate only — your actual cost depends on your insurance plan. Always verify pricing with your pharmacy or insurer.

Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.