Gaucher disease

US FDA approves velaglucerase alfa for injection to treat rare genetic disorder Gaucher disease

The US Food and Drug Administration has approved velaglucerase alfa for injection (VPRIV) to treat children and adults with a form of the rare genetic disorder Gaucher disease.

Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. Without this enzyme, harmful amounts of a certain fatty substance (lipid) can build up in the liver, spleen, bones, bone marrow and nervous system, and can prevent cells and organs from working properly. About one in 50,000 to one in 100,000 people in the general population have Gaucher disease.