Alglucosidase alfa Drug Aliquot | Anti Lysosomal glycogen | Therapeutic Enzyme

Aliquoted from the original reference drug | Now available for research use

Aliquots of the original Alglucosidase alfa (Myozyme®) are now available as research consumables.

Alglucosidase alfa drug aliquots can be used for in vitro or in vivo experiments. Several other reference drugs are also available.

The drug aliquots are generated by aliquoting (dividing and repackaging) the EU-licensed reference drug.

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Myozyme® / Alglucosidase alfa Reference Product

Drug nameMyozyme®
INNAlglucosidase alfa
API typeEnzyme
Pharmacotherapeutic group
Other alimentary tract and metabolism products, enzymes
ATC code
A16AB07
Target of antibody
Lysosomal glycogen
General function
Short descriptionAlglucosidase alfa is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency). Alglucosidase alfa is indicated in adults and paediatric patients of all ages.
Pharmacodynamic properties
(Mechanism of action; Source EMA document)
It is postulated that Myozyme will restore lysosomal GAA activity resulting in stabilisation or restoration of cardiac and skeletal muscle function (including respiratory muscles). Due to the blood-brain barrier effect and the enzyme’s size, uptake of alglucosidase alfa in the central nervous system is
unlikely.
Pharmacodynamic properties (Pharmacodynamic effects; Source EMA document)Pompe disease is a rare, progressive and fatal metabolic myopathy with an estimated global incidence of 1 in 40,000 births. Other names for Pompe disease include glycogen storage disease type II (GSD-II), acid maltase deficiency (AMD) and glycogenosis type II. Pompe disease belongs to the lysosomal storage disorders as it is caused by a deficiency of a naturally occurring lysosomal hydrolase, acid α-glucosidase (GAA) that degrades lysosomal glycogen to glucose. Deficiency of this enzyme leads to glycogen accumulation in various tissues, particularly cardiac, respiratory and skeletal muscle, leading to the development of hypertrophic cardiomyopathy and progressive muscle weakness, including impairment of respiratory function.
Original license holder
Sanofi B.V.
Paasheuvelweg 25
1105 BP Amsterdam
The Netherlands
Marketing authorisation numbers
EU/1/06/333/001-003
Marketing authorisation holder
Sanofi B.V.
Paasheuvelweg 25
1105 BP Amsterdam
The Netherlands
Name of the manufacturer of the biological active substance
Genzyme Corporation 45, 51, 76, 74 and 80 New York Avenue, Framingham, MA 01701, United States

Genzyme Flanders, Cipalstraat 8, 2440 Geel, Belgium
Name and address of the manufacturer(s) responsible for batch releaseGenzyme Ireland Limited, IDA Industrial Park, Old Kilmeaden Road, Waterford, Ireland
Max shelf life
3 years
Storage conditions
2°C – 8°C
List of excipients
Mannitol (E421)
Sodium dihydrogen phosphate monohydrate (E339)
Disodium phosphate heptahydrate (E339)
Polysorbate 80 (E433)

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Evidentic offers multiple batches of original drug aliquots in low binding Eppendorf tubes for research use only. We provide licence-free drug aliquots with long, short or even “negative shelf life” by storing the products at recommended temperatures (either 2-8°C or -80°C) and ensuring fully functional molecules for research purposes.