FDA Approves New Gaucher Therapy

28 February 2010 (Last Updated February 28th, 2010 18:30)

The US Food and Drug Administration (FDA) has approved velaglucerase alfa for injection (VPRIV), a new treatment alternative for patients with the rare genetic disorder Gaucher disease. People who suffer from Gaucher disease do not produce enough of an enzyme called glucocerebrosidase, wh

The US Food and Drug Administration (FDA) has approved velaglucerase alfa for injection (VPRIV), a new treatment alternative for patients with the rare genetic disorder Gaucher disease.

People who suffer from Gaucher disease do not produce enough of an enzyme called glucocerebrosidase, which allows harmful amounts of a certain fatty substance (lipid) that 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 US population have Gaucher disease.

The new injection VPRIV, manufactured by Shire, provides long-term enzyme replacement therapy for Type 1 Gaucher disease, the most common form of the genetic disorder. The drug is an alternative to Cerezyme (imiglucerase), another enzyme replacement therapy, which is in short supply.

The FDA's Office of Drug Evaluation III director Julie Beitz said that patients who previously received Cerezyme as an enzyme replacement therapy for their Type 1 Gaucher disease can be safely switched to VPRIV.

The safety and effectiveness of VPRIV was assessed during three clinical studies involving 82 patients with Type 1 Gaucher disease aged four years and older. The studies included patients who switched to VPRIV after being treated with Cerezyme.