Avalglucosidase 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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Up to 10 batches of original drugs for examining batch-to-batch variations
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Up to 80% less compared to the original pharmaceutical price
| Drug name | Nexviadyme® |
| INN | Avalglucosidase alfa |
| API type | Enzyme |
| Pharmacotherapeutic group | Not assigned yet |
| ATC code | Not assigned yet |
| Target of antibody | Lysosomal glycogen |
| General function | |
| Short description | Avalglucosidase alfa is indicated for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). |
| Pharmacodynamic properties (Mechanism of action; Source EMA document) | Avalglucosidase alfa is a recombinant human acid α-glucosidase (rhGAA) that provides an exogenous source of GAA. Avalglucosidase alfa is a modification of alglucosidase alfa in which approximately 7 hexamannose structures each containing 2 terminal mannose-6-phosphate (bis-M6P) moieties are conjugated to oxidized sialic acid residues on alglucosidase alfa. Avalglucosidase alfa has a 15-fold increase in mannose-6-phosphate (M6P) moieties compared with alglucosidase alfa. Binding to M6P receptors on the cell surface has been shown to occur via carbohydrate groups on the GAA molecule, after which it is internalised and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity to degrade glycogen. |
| Pharmacodynamic properties (Pharmacodynamic effects; Source EMA document) | Avalglucosidase alfa is a recombinant alpha-glucosidase (GAA) enzyme that catalyzes hydrolysis of glycogen. In clinical trials, avalglucosidase alfa reduced the levels of glycogen excreted in the urine of patients with Pompe disease, indicating that it effectively cleaved excess glycogen. Avalglucosidase alfa has significantly higher binding affinity for cation-independent mannose-6-phosphate receptor (CI-MPR) for cellular uptake and better muscle targeting than alglucosidase alfa. In GAA-deficient mice, avalglucosidase alfa reduced glycogen with more efficacy than alglucosidase alfa at an equivalent dose. |
| Original license holder | Sanofi B.V. Paasheuvelweg 25 1105 BP Amsterdam The Netherlands |
| Marketing authorisation numbers | EU/1/21/1579/001-4 |
| Marketing authorisation holder | Sanofi B.V. Paasheuvelweg 25 1105 BP Amsterdam The Netherlands |
| Name of the manufacturer of the biological active substance | Genzyme Flanders Cipalstraat 8 2440 Geel, Belgium |
| Name and address of the manufacturer(s) responsible for batch release | Genzyme Ireland Limited, IDA Industrial Park, Old Kilmeaden Road, Waterford, Ireland |
| Max shelf life | 4 years |
| Storage conditions | 2°C – 8°C |
| List of excipients | Histidine Histidine hydrochloride monohydrate Glycine Mannitol Polysorbate 80 |
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.
Evidentic GmbH
Am Mühlenberg 10
14476 Potsdam | Germany
