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FDA Approves Therapy to Treat Gaucher Disease

New drug will offer a treatment alternative for patients with rare genetic disorder

The U.S. 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 1 in 50,000 to 1 in 100,000 people in the general population have Gaucher disease.

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 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.

SOURCE:

U.S. Food and Drug Administration



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