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FDA Approves VPRIV to Treat Gaucher Disease
Posted 28 Feb 2010 by Drugs.com
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 ... Read more
Related support groups: Gaucher Disease
Replacement Therapy Approved for Gaucher Disease
Posted 27 Feb 2010 by Drugs.com
FRIDAY, Feb. 26 – Velaglucerase alfa for injection (VPRIV) has been approved by the U.S. Food and Drug Administration to treat a rare inherited disorder called Gaucher disease, the agency said Friday. The disorder is caused by lack of an enzyme called glucocerebrosidase. This allows a fatty substance called a lipid to build up in the liver, spleen, bones, bone marrow and nervous system. The disorder affects only about 1 in 50,000 people, the FDA said in a news release. The newly approved treatment replaces the lacking enzyme in people with Type 1 Gaucher disease, the disorder's most common form. An older form of replacement therapy is in short supply, the agency said. VPRIV's safety and effectiveness were evaluated in clinical trials involving 82 people aged 4 and older with Type 1 Gaucher. The most common side effects reported were allergic reactions; dizziness; pain in the abdomen, ... Read more
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