Velaglucerase alfa is a prescription medication used to treat a rare genetic disorder called Gaucher disease. It is a man-made form of an enzyme, glucocerebrosidase, which is produced naturally in the body. This medication helps to reduce the signs and symptoms of Gaucher disease by helping to break down a fatty substance called glucocerebroside that is normally broken down by the enzyme. It is given as a subcutaneous injection (under the skin) into the abdomen, thigh, or upper arm. Common side effects of Velaglucerase alfa include injection site reactions and mild to moderate flu-like symptoms.
Velaglucerase alfa, sold under the brand name Vpriv, is a medication used for the treatment of Gaucher disease type 1 [1]. Here's a closer look at this enzyme replacement therapy:
Function:
- Replaces a missing enzyme: Gaucher disease type 1 is caused by a deficiency in the enzyme glucocerebrosidase. Velaglucerase alfa acts as an artificial replacement for this enzyme.
Mechanism of action:
- Breaks down harmful substances: The missing enzyme in Gaucher disease leads to a buildup of a fatty substance called glucocerebroside in various organs. Velaglucerase alfa works by breaking down this harmful substance, alleviating the symptoms of the disease.
Classification:
- Enzyme replacement therapy (ERT)
- Recombinant human glucocerebrosidase: It's an engineered version of the natural enzyme, identical in amino acid sequence.
Administration:
- Intravenous infusion (IV drip): Typically administered every other week, taking up to 60 minutes for the infusion [4].