Cerezyme

What is Cerezyme?

Cerezyme is a powder that is made up into a solution for infusion (a drip into a vein). It contains the active substance imiglucerase. It is used for the treatment of Gaucher disease.

 

What is Cerezyme used for?

Cerezyme 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 acid beta-glucosidase, which normally breaks down a fatty waste product called glucosylceramide. Without the enzyme, glucosylceramide builds up in the body, typically in the liver, spleen and bone marrow, 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.
Cerezyme is used in patients who have type 1 Gaucher disease, which does not affect the nerve cells, or type 3 Gaucher disease, which progresses slowly and affects the nerve cells. The patients must have symptoms that are not affecting the nervous system, including one or more of the following conditions:

• anaemia;
• thrombocytopenia (low blood platelet counts);
• bone disease;
• an enlarged liver or spleen.

The medicine can only be obtained with a prescription.

 

How is Cerezyme used?

Patients with Gaucher disease should be managed by doctors who are knowledgeable about the disease’s treatment. Cerezyme is usually given by infusion every two weeks. The dose and how often the infusions are given need to be adjusted according to each individual patient’s symptoms and response to treatment. The first few infusions should be given slowly, but after these, the speed of infusion can be increased under the supervision of a doctor or nurse. After training, the patient or carer can give the infusion at home, if their doctor believes it is appropriate.

How does Cerezyme work?

Gaucher disease has previously been treated using an enzyme called alglucerase, which was prepared from human placentas. Imiglucerase, the active substance in Cerezyme, is a copy of this enzyme, which is produced by a method known as ‘recombinant DNA technology’: the enzyme is made by a cell that has received a gene (DNA), which enables it to produce the enzyme. Imiglucerase replaces the missing enzyme in Gaucher disease, helping to break down glucosylceramide and stopping it building up in the body.

Source: European Medicines Agency