Miglustat G.L.

Miglustat G.L. contains the active substance miglustat, which belongs to a group of medicines that affects metabolism. It is used to treat two diseases:

• Miglustat G.L. is used to treat mild to moderate Gaucher disease type 1 in adults.

In Gaucher disease type 1, a substance called glucosylceramide is not removed from your body. It accumulates in certain cells in your immune system. This can lead to enlargement of the liver and spleen, blood changes, and bone disease.

The usual treatment for Gaucher disease type 1 is enzyme replacement therapy. Miglustat G.L. is used only in patients for whom enzyme replacement therapy is not an option.

• Miglustat G.L. is also used in Niemann-Pick disease type C to treat progressive neurological symptoms in adults and children.

If you have Niemann-Pick disease type C, fats, such as glycosphingolipids, accumulate in the cells of your brain. This can lead to disturbances in neurological functions such as slow eye movements, balance, swallowing, and memory, as well as seizures.

The active principle of Miglustat G.L. is the inhibition of an enzyme called “glucosylceramide synthase”. It is responsible for the first step in the synthesis of most glycosphingolipids.