A medication used to treat a rare genetic disorder called Gaucher disease, which is caused by the deficiency of a specific enzyme in the body.


An enzyme replacement therapy used for the long-term treatment of for pediatric and adult patients with type 1 Gaucher disease.


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.



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.


Information currently not available.

Mechanism of action

Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside.


Information currently not available.

Protein binding

Information currently not available.

Volume of distribution

The mean volume of distribution at steady state ranges from 82 to 108 mL/kg (8.2% to 10.8% of body weight).


Mean clearance ranges from 6.72 to 7.56 mL/min/kg.

Half life

11-12 minutes.

Route of elimination

Information currently not available.


Information currently not available.

Adverse Effects


Information currently not available.

Food Interactions

    Information currently not available.


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2 References
  1. 1 . 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: 20336596
  2. 2 . Vairo F, Netto C, Dorneles A, Mittelstadt S, Wilke M, Doneda D, Michelin K, Ribeiro CB, Quevedo A, Vieira T, Nalin T, Lueska S, Schwartz IV: Enzyme Replacement Therapy in a Patient with Gaucher Disease Type III: A Paradigmatic Case Showing Severe Adverse Reactions Started a Long Time After the Beginning of Treatment. JIMD Rep. 2013;11:1-6. doi: 10.1007/8904_2013_214. Epub 2013 Feb 21.PubMed: 23430813