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

Gaucher's disease

Gaucher's disease is a genetic disease in which a fatty substance accumulates in cells and certain organs.Gaucher's disease is the most common of the lysosomal storage diseases. It is caused by a hereditary deficiency of the enzyme glucosylceramidase. The enzyme acts on the fatty acid...

.

It is a recombinant DNA

Recombinant DNA

Recombinant DNA molecules are DNA sequences that result from the use of laboratory methods to bring together genetic material from multiple sources, creating sequences that would not otherwise be found in biological organisms...

-produced analogue

Analog (chemistry)

In chemistry, a structural analog , also known as chemical analog or simply analog, is a compound having a structure similar to that of another one, but differing from it in respect of a certain component. It can differ in one or more atoms, functional groups, or substructures, which are replaced...

 of human β-glucocerebrosidase

Glucocerebrosidase

β-Glucocerebrosidase is an enzyme with glucosylceramidase activity that is needed to cleave, by hydrolysis, the beta-glucosidic linkage of the chemical glucocerebroside, an intermediate in glycolipid metabolism...

.
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

Gaucher's disease

Gaucher's disease is a genetic disease in which a fatty substance accumulates in cells and certain organs.Gaucher's disease is the most common of the lysosomal storage diseases. It is caused by a hereditary deficiency of the enzyme glucosylceramidase. The enzyme acts on the fatty acid...

. 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

Orphan drug

An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease...

 status in the USA, Australia, and Japan.

Cerezyme was one of the drugs manufactured at Genzyme's Allston, Massachusetts plant, for which production was disrupted in 2009 after contamination with Vesivirus 2117.

See also

• Other drugs for the treatment of Gaucher's disease
  • Isofagomine tartrate
    Isofagomine tartrate
    Isofagomine tartrate is an experimental drug for the treatment of certain forms of Gaucher's disease, developed by Amicus Therapeutics and Shire plc....
    
    
  • Miglustat
    Miglustat
    Miglustat is a drug developed by and is used primarily to treat Type 1 Gaucher disease . It is marketed under the trade name Zavesca. Miglustat is an imino sugar , a synthetic analogue of D-glucose and a white to off-white crystalline solid that has a bitter taste...