Velaglucerase Alpha

Table of contents

  • Brand Names
  • Chemistry
  • Pharmacologic Category
  • Mechanism of Action
  • Therapeutic Use
  • Pregnancy and Lactation Implications
  • Contraindications
  • Warnings and Precautions
  • Adverse Reactions
  • Genes that may be involved
  • Drug Interactions
  • Dosage
  • Pharmacokinetics and Pharmacodynamics

Brand Names

Europe

Austria: VPRIV; Bulgaria: VPRIV; Czech Republic: VPRIV; Denmark: VPRIV; Estonia: VPRIV; France: VPRIV; Germany: VPRIV; Ireland: VPRIV; Latvia: VPRIV; Lithuania: VPRIV; Luxembourg: VPRIV; Malta: VPRIV; Netherlands: VPRIV; Poland: VPRIV; Portugal: VPRIV; Slovakia: VPRIV; Slovenia: VPRIV; Sweden: VPRIV; UK: VPRIV.

North America

Canada: VPRIV; USA: VPRIV.

Drug combinations

Chemistry

Velagulcerase Alpha: C~2532~H~3854~N~672~O~711~S~16~. 63 kDa. (1) Ceramidase, glucosyl-(human HT-1080 cell); (2) Human glucosylceramidase (EC 3.2.1.45 or β-glucocerebrosidase), glycoform α. Glycoprotein of 497 amino acids, with the same amino acid sequence as the naturally-occurring human enzyme, glucocerebrosidase. Contains 5 potential N-linked glycosylation sites; four of these sites are occupied by glycan chains. Manufactured to contain predominantly high mannose-type N-linked glycan chains. CAS-884604-91-5.

Pharmacologic Category

Enzymes. Hydrolytic Lysosomal Glucocerebroside-Specific Enzyme. (ATC-Code: A16AB10).

Mechanism of action

Gaucher’s disease is an autosomal recessive disorder caused by mutations in the GBA gene, which results in a deficiency of the lysosomal enzyme β-glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucocerebroside primarily in the lysosomal compartment of macrophages, giving rise to foam cells or «Gaucher cells». In this lysosomal storage disorder (LSD), clinical features are reflective of the accumulation of Gaucher cells in the liver, spleen, bone marrow, and other organs. The accumulation of Gaucher cells in the liver and spleen leads to organomegaly. The presence of Gaucher cells in the bone marrow and spleen leads to clinically significant anemia and thrombocytopenia. Velaglucerase alpha catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside.

Therapeutic use

Indicated for long-term enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher’s disease.

Pregnancy and lactiation implications

Should be used during pregnancy only if clearly needed. Not recommended during lactation.

Unlabeled use

Contraindications

None known to date.

Warnings and precautions

Hypersensitivity reactions to the product have been reported. Infusion-related reactions were the most commonly observed adverse reactions in patients treated with velaglucerase.

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