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Gaucher Disease News

FDA Approves Cerdelga (eliglustat) for Type 1 Gaucher Disease

Posted 19 Aug 2014 by Drugs.com

August 19, 2014 – The U.S. Food and Drug Administration today approved Cerdelga (eliglustat) for the long-term treatment of adult patients with the Type 1 form of Gaucher disease, a rare genetic disorder. Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials to collect in the spleen, liver and bone marrow. The major signs of Gaucher disease include liver and spleen enlargement, low red blood cell counts (anemia), low blood platelet counts and bone problems. Cerdelga is a hard gelatin capsule containing eliglustat that is taken orally. In patients with Gaucher disease Type 1, the drug slows down the production of the fatty materials by inhibiting the metabolic process that forms them. Type 1 Gaucher disease is estimated to affect about 6,000 people in the United States. “Today’s approval offers a ... Read more

Related support groups: Gaucher Disease

FDA Medwatch Alert: VPRIV (velaglucerase alfa for injection): Recall - Visible Particulate Matter

Posted 20 Mar 2014 by Drugs.com

ISSUE: Shire Pharmaceuticals announced a voluntary recall in the United States of one batch, packaged into three lots, of VPRIV due to the presence of visible particulate matter, identified as stainless steel and barium sulfate. The particulate matter was found in a small number of vials in the three packaged lots of VPRIV. A Shire investigation identified the particulate matter root cause as the third party supplier fill finish process. If infused, there is a possibility of rare but serious adverse events associated with particulate containing barium sulfate. The following packaged lots were recalled: FEW13-001, FEW13-002, and FED13-006. These lots were distributed nationwide to hospitals, infusion clinics, patients, and home health agencies in the United States and all have the same NDC code (54092-701-04) and same expiration date of 10/15 (Oct 2015). BACKGROUND: VPRIV is a hydrolytic ... Read more

Related support groups: Gaucher Disease, VPRIV

FDA Approves New Orphan Drug Elelyso to Treat a Form of Gaucher Disease

Posted 1 May 2012 by Drugs.com

May 1, 2012 – The U.S. Food and Drug Administration today approved Elelyso (taliglucerase alfa) for long-term enzyme replacement therapy to treat a form of Gaucher disease, a rare genetic disorder. Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials (lipids) to collect in the spleen, liver, kidneys, and other organs. The major signs of Gaucher disease include liver or spleen damage, low red blood cell counts (anemia), low blood platelet counts, and bone problems. Elelyso is an injection that replaces the missing enzyme in patients with a confirmed diagnosis of Type 1 (non-neuropathic) Gaucher disease and should be administered by a health care professional every other week. Type 1 Gaucher disease is estimated to affect about 6,000 people in the United States. "Today's approval provides for a ... Read more

Related support groups: Gaucher Disease

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

Related support groups: Gaucher Disease

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Related Condition Support Groups

Cerebral Sphingolipidosis, Metabolic Disorder Including Congenital

Related Drug Support Groups

Zavesca, Cerezyme, miglustat, VPRIV, Cerdelga, Elelyso, eliglustat, velaglucerase alfa, taliglucerase alfa, imiglucerase