Vpriv

Vpriv is a powder that is made up into a solution for infusion (a drip into a vein). It contains the active substance velaglucerase alfa and is used to treat Gaucher disease.

What is Vpriv used for?

Vpriv is used for the long-term treatment of patients with Gaucher disease. Gaucher disease is a rare inherited disorder, in which people do not have enough of an enzyme called glucocerebrosidase, which normally breaks down a fat called glucocerebroside. Without the enzyme, glucocerebroside builds up in the body, typically in the liver, spleen and bone, which produces the symptoms of the disease: anaemia (low red-blood-cell counts), tiredness, easy bruising and a tendency to bleed, an enlarged spleen and liver, and bone pain and breaks.
Vpriv is used in patients who have type-1 Gaucher disease, the type that usually affects the liver, spleen and bones.
Because the number of patients with Gaucher disease is low, the disease is considered ‘rare’, and Vpriv was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 9 June 2010.
The medicine can only be obtained with a prescription.

How is Vpriv used?

Vpriv treatment should be supervised by a doctor experienced in managing Gaucher disease.
Vpriv is given as a one‑hour infusion once every two weeks. The dose can be adjusted according to each patient’s symptoms and response to treatment. The first three infusions are given in hospital, but subsequent infusions given at home administration can be considered in patients who tolerate the medicine well. Home infusions should be supervised by a healthcare professional.

How does Vpriv work?

Gaucher disease occurs because of the lack of an enzyme called glucocerebrosidase. The active substance in Vpriv, velaglucerase alfa, is a copy of this enzyme, which is produced by a method known as ‘recombinant DNA technology’: the enzyme is made by activating a gene in a human cell that makes it able to produce this enzyme. Velaglucerase alfa replaces the missing enzyme in Gaucher disease, helping to break down glucocerebroside and stopping it building up in the body.

How has Vpriv been studied?

The effects of Vpriv were first tested in experimental models before being studied in humans.
In one main study involving 35 patients (including 9 children) with type-1 Gaucher disease, Vpriv was compared with imiglucerase (another medicine for Gaucher disease). The main measure of effectiveness was the improvement in anaemia, one of the symptoms of the disease, after 41 weeks. The study also looked at control of other signs of the disease such as the increase in the number of platelets in the blood, and the reduction of the size of the liver and spleen.

What benefit has Vpriv shown during the studies?

Vpriv was as effective as imiglucerase at reducing anaemia. Vpriv increased the amount of haemoglobin (the protein in red blood cells that carries oxygen) by an average of 1.6 grams per decilitre (from 11.4 g/dl) while imiglucerase increased the amount of haemoglobin by an average of 1.5 g/dl (from 10.6 g/dl). The study also showed that Vpriv is as effective as imiglucerase in controlling other symptoms of Gaucher disease.

What is the risk associated with Vpriv?

In studies, the most common side effects with Vpriv (seen in more than 1 patient in 10) were headache, dizziness, bone pain, arthralgia (joint pain), back pain, infusion-related reactions, asthenia (weakness) or fatigue (tiredness), and pyrexia (fever) or increased body temperature. For the full list of all side effects reported with Vpriv, see the package leaflet.
Vpriv must not be used in people who have a severe allergic reaction to velaglucerase alfa or any of the other ingredients.

Source: European Medicines Agency