Description

Simple

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.

Clinical

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

Overview

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.

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.

Pharmacodynamic

Information currently not available.

Mechanism of action

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

Absorption

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).

Clearance

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

Half life

11-12 minutes.

Route of elimination

Information currently not available.

Toxicity

Information currently not available.

Adverse Effects

Contraindications

Information currently not available.

Food Interactions

    Information currently not available.

Interactions

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  • Paracetamol(acetaminophen)
  • Paxil(paroxetine)
  • Pamelor(nortriptyline)
  • Panadol(acetaminophen)
  • Patanol(olopatadine ophthalmic)
  • Pataday(olopatadine ophthalmic)
  • Parnate(tranylcypromine)
  • Pazeo(olopatadine ophthalmic)
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