FDA Approves VPRIV for Treatment in Gaucher's Disease


On February 26 FDA approved velaglucerase alfa for injection (VPRIV) to treat children and adults with a form of the rare genetic disorder Gaucher disease.

Gaucher disease is an autosomal recessive lysosomal storage disorder caused by mutations in the glucocerebrosidase gene. It affects about 1 in 50,000 to 1 in 100,000 people in the general population. A person with Gaucher disease is unable to produce enough of the enzyme glucocerebrosidase. Without this enzyme, harmful amounts of a lipid build up in the liver, spleen, bones, bone marrow and nervous system, preventing cells and organs from working properly.

VPRIV provides long-term enzyme replacement therapy for Type 1 Gaucher disease which is the most common form of the genetic disorder. VPRIV will be an alternative enzyme replacement therapy to Cerezyme (imiglucerase). Cerezyme is currently in short supply.

The safety and effectiveness of VPRIV was assessed in three clinical studies involving 82 patients with Type 1 Gaucher disease ages 4 years and older. The studies included patients who switched to VPRIV after being treated with Cerezyme.

The most common adverse reactions to VPRIV are allergic reactions. Other observed adverse reactions with VPRIV are headache, dizziness, abdominal pain, back pain, joint pain, nausea, fatigue/weakness, fever, and prolongation of activated partial thromboplastin time, a measure of clotting time.
VPRIV is manufactured by Shire Human Genetic Therapies Inc. of Cambridge, Mass.


Three types of Gaucher disease

Type 1 is by far the most common. Patients in this group usually bruise easily and experience fatigue due to anemia and low blood platelets. They also have an enlarged liver and spleen, skeletal disorders, and, in some instances, lung and kidney impairment. There are no signs of brain involvement. Symptoms can appear at any age.

Type 2 Gaucher disease patients have liver and spleen enlargement which are apparent by 3 months of age. Patients have extensive and progressive brain damage and usually die by 2 years of age.

Type 3 Guacher patients will have variable liver and spleen enlargement. Signs of brain involvement such as seizures gradually become apparent.

For more information:
National Human Genome Research Institute – Learning About Gaucher Disease
National Institute of Neurological Disease and Stroke – Gaucher’s Disease Information

Food and Drug Administration

This page is updated on May 12, 2013.