Velmanase Alfa
Velmanase alfa is a recombinant human lysosomal alpha-mannosidase produced in Chinese hamster ovary cells for enzyme replacement therapy (ERT) in patients with alpha-mannosidosis, a rare lysosomal storage disease caused by deficiency of the alpha-mannosidase enzyme. It catalyzes the hydrolysis of mannose-containing oligosaccharides in lysosomes, preventing their pathological accumulation in cells. Regular intravenous infusions reduce mannose-rich oligosaccharide levels and improve clinical manifestations of the disease.
Moleküler Ağırlık
65,4000 g/mol
TPSA
0,00 Ų
Etki Mekanizması
As a recombinant protein, it replicates the function of the naturally occurring human protein, supplementing or replacing deficient endogenous production to restore normal physiological activity.
Pharmacokinetics (PK)
Pharmacodynamics (PD)
As a recombinant protein, it replicates the function of the naturally occurring human protein, supplementing or replacing deficient endogenous production to restore normal physiological activity.
SMILES
[Zn+2]
InChI
InChI=1S/Zn/q+2
Molecular Formula
Zn+2
HBD / HBA
- / -
Döndürülebilir Bağlar
0
Ağır Atomlar
1
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
Sıkça Sorulan Sorular
Velmanase alfa is a recombinant human lysosomal alpha-mannosidase produced in Chinese hamster ovary cells for enzyme replacement therapy (ERT) in patients with alpha-mannosidosis, a rare lysosomal storage disease caused by deficiency of the alpha-mannosidase enzyme. It catalyzes the hydrolysis of mannose-containing oligosaccharides in lysosomes, preventing their pathological accumulation in cells. Regular intravenous infusions reduce mannose-rich oligosaccharide levels and improve clinical manifestations of the disease.
As a recombinant protein, it replicates the function of the naturally occurring human protein, supplementing or replacing deficient endogenous production to restore normal physiological activity.
Yes, Velmanase Alfa is an approved drug. It has reached clinical phase 4. It is classified as a Enzyme.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4298010. Open-access bioactivity database.
- PubChem — National Center for Biotechnology Information (NCBI). CID 32051. Chemical information database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-28.
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