Imiglucerase

Imiglucerase is a medication used in the treatment of Gaucher's disease.[2][3]

It is a recombinant DNA-produced analogue of the human enzyme β-glucocerebrosidase. Cerezyme is a freeze-dried medicine containing imiglucerase, manufactured by Genzyme Corporation. It is given intravenously after reconstitution as a treatment for Type 1 and Type 3[4] Gaucher's disease. It is available in formulations containing 200 or 400 units per vial. The specific activity of highly purified human enzyme is 890,000 units/mg,[5] meanwhile the enzyme activity produced by recombinant DNA technology is approximately 40 units/mg.[6] A typical dose is 2.5U/kg every two weeks, up to a maximum of 60 U/kg once every two weeks, and safety has been established from ages 2 and up.[7] It is one of more expensive medications, with an annual cost of $200,000 per person in the United States.[8] Imiglucerase has been granted orphan drug status in the United States, Australia, and Japan.[9]

Cerezyme was one of the drugs manufactured at Genzyme's Allston, Massachusetts plant, for which production was disrupted in 2009 after contamination with Vesivirus 2017.[10]

Side effects

The most common side effect is hypersensitivity, which occurs in about 3% of patients. It is associated with symptoms such as cough, shortness of breath, rashes, itching, and angiooedema. Less common side effects include dizziness, headache, nausea, diarrhea, and reactions at the injection site; they are found in less than 1% of patients.[4]

Interactions

No clinical interaction studies have been conducted.[4] Miglustat appears to increase the clearance of imiglucerase by 70%, resulting in decreased enzyme activity.[11]

See also

• Other drugs for the treatment of Gaucher's disease
• Afegostat (development terminated)
• Eliglustat
• Miglustat
• Velaglucerase alfa
• taliglucerase alfa