Imiglucerase

Imiglucerase is a medication used in the treatment of Gaucher's disease.

It is a recombinant DNA-produced analogue of human 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 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. 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. It is one of the most expensive drugs sold, with an annual cost to U.S. patients of $200,000. Due to the low profitability (and high expense) of developing medications for rare conditions, imiglucerase has been granted orphan drug status in the USA, Australia, and Japan.