Identification
Name Velaglucerase alfa
Accession Number DB06720
Type biotech
Description Velaglucerase alfa is a gene-activated human recombinant glucocerebrosidase used for the treatment of Type 1 Gaucher disease, caused by a deficiency of the lysosomal enzyme glucocerebrosidase. Additionally, Velaglucerase alfa has also been investigated for use in Type 3 Gaucher disease.
Structure
Categories (*)
Molecular Weight ~63 kDa
Groups approved
Monoisotopic Weight Not Available
Pharmacology
Indication Velaglucerase alfa is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy for pediatric and adult patients with type 1 Gaucher disease.
Mechanism of action Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside.
Absorption Not Available
Protein binding Not Available
Biotransformation Not Available
Route of elimination Not Available
Toxicity Not Available
Affected organisms Not Available
Interactions
Drug Interactions Not Available
Food Interactions Not Available
Glucosylceramidase
Name Glucosylceramidase
Gene Name GBA
Pharmacological action unknown
Actions Not Available
References
  • Pastores GM: Velaglucerase alfa, a human recombinant glucocerebrosidase enzyme replacement therapy for type 1 Gaucher disease. Curr Opin Investig Drugs. 2010 Apr;11(4):472-8. - Pubmed
DTHybrid score 1.1806