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Myozyme (Genzyme): Full Drug Profile

Medically reviewed by Min Clinic Staff | Updated: January 2026

Myozyme (Genzyme) - General Information

Myozyme (Genzyme) consists of the human enzyme acid a-glucosidase (GAA), encoded by the most predominant of nine observed haplotypes of this gene. Myozyme (Genzyme) is produced by recombinant DNA technology in a Chinese hamster ovary cell line. Myozyme (Genzyme) degrades glycogen by catalyzing the hydrolysis of a-1,4- and a-1,6- glycosidic linkages of lysosomal glycogen.

 

Pharmacology of Myozyme (Genzyme)

Pompe disease (glycogen storage disease type II, GSD II, glycogenosis type II, acid maltase deficiency) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA. In the infantile-onset form, Pompe disease results in intralysosomal accumulation of glycogen in various tissues, particularly cardiac and skeletal muscles, and hepatic tissues, leading to the development of cardiomyopathy, progressive muscle weakness, and impairment of respiratory function. In the juvenile- and adult-onset forms, intralysosomal accumulation of glycogen is limited primarily to skeletal muscle, resulting in progressive muscle weakness. Death in all forms is usually related to respiratory failure. Myozyme (Genzyme) provides an exogenous source of GAA. Binding to mannose-6-phosphate receptors on the cell surface has been shown to occur via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen.

 

Myozyme (Genzyme) for patients

Patients and their caregivers should be informed that a registry for patients with Pompe disease has been established in order to better understand the variability and progression of Pompe disease and to continue to monitor and evaluate treatments. Patients and their caregivers are encouraged to participate and should be advised that their participation may involve long-term follow-up. Information regarding the registry program may be found at www.pomperegistry.com or by calling 1-800-745-4447.

 

Myozyme (Genzyme) Interactions

No drug interaction studies have been performed.

 

Myozyme (Genzyme) Contraindications

 

Additional information about Myozyme (Genzyme)

Myozyme (Genzyme) Indication: For the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients. Mechanism Of Action: Myozyme (Genzyme) is designed to act as an exogenous source of GAA, acting to correct GAA deficiency that is the hallmark of Pompe disease. Myozyme (Genzyme) binds to mannose-6-phosphate receptors on the cell surface via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen. Drug Interactions: Not Available Food Interactions: Not Available Generic Name: Alglucosidase alfa Synonyms: Not Available Drug Category: Enzyme Replacement Agents Drug Type: Biotech; Approved Other Brand Names containing Alglucosidase alfa: Myozyme (Genzyme); Absorption: Not Available Toxicity (Overdose): There have been no reports of overdose with alglucosidase alfa. Protein Binding: Not Available Biotransformation: Not Available Half Life: 2.3 ± 0.4 hours. Dosage Forms of Myozyme (Genzyme): Powder, for solution IntravenousPowder, for solution Intravenous Chemical IUPAC Name: Not Available Chemical Formula: C4758H7262N1274O1369S35 Alglucosidase alfa on Wikipedia: https://en.wikipedia.org/wiki/Alglucosidase_alfa Organisms Affected: Humans and other mammals