Gaucher Disease Therapy (VPRIV) Approved by FDA

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Gaucher Disease

Gaucher's disease or Gaucher disease (GD) is a genetic disorder in which glucocerebroside (a sphingolipid, also known as glucosylceramide) accumulates in cells and specific organs. The condition is characterized by bruising, fatigue, anemia, low blood platelet count, and liver and spleen enlargement. It is caused by a hereditary deficiency of the enzyme glucocerebrosidase (also known as glucosylceramidase), which acts on the glucocerebroside. Type 1 Gaucher disease is the most common form of this condition. Type 1 is also called non-neuronopathic Gaucher disease because the brain and spinal cord (the central nervous system) are usually not affected. Types 2 and 3 of Gaucher disease are known as neuronopathic forms of the disorder because they are characterized by problems that affect the central nervous system. In addition to the signs and symptoms described above, these conditions can cause abnormal eye movements, seizures, and brain damage. The most severe type of Gaucher disease is called the perinatal lethal form. This condition causes severe or life-threatening complications starting before birth or in infancy. Another form of Gaucher disease is the cardiovascular type because it primarily affects the heart, causing the valves to harden (calcify).

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FDA Approves Therapy to Treat Gaucher Disease - New drug will offer a treatment alternative for patients with rare genetic disorder.

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 prevent cells and organs from working properly. About 1 in 50,000 to 1 in 100,000 people in the general population have Gaucher disease.

Velaglucerase alfa for injection (VPRIV) provides long-term enzyme replacement therapy for Type 1 Gaucher disease, the most common form of the genetic disorder. It is an alternative to Cerezyme (imiglucerase), another enzyme replacement therapy. Cerezyme is currently in short supply.

"The approval of VPRIV will provide a safe and effective alternative treatment for patients with Gaucher disease," said Julie Beitz, M.D., director of the FDA's Office of Drug Evaluation III. "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 were assessed in three clinical studies involving 82 patients with Type 1 Gaucher disease ages four 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 prolonged activated partial thromboplastin time, a measure of clotting time.

VPRIV is manufactured by Shire Human Genetic Therapies Inc. of Cambridge, Mass.

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