Gaucher Disease Therapy Approved by FDA
Author: U.S. Food and Drug Administration
Peer-Reviewed Publication: N/A
Synopsis: New drug will offer a treatment alternative for patients with rare genetic Gaucher Disease. FDA Approves Therapy to Treat Gaucher Disease - New drug will offer a treatment alternative for patients with rare genetic disorder.
FDA Approves Therapy to Treat Gaucher Disease - New drug will offer a treatment alternative for patients with rare genetic disorder.
This article is from our digest of publications relating to Pharmaceutical Information that also includes:
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.
For more information
National Human Genome Research Institute - Learning About Gaucher Disease
National Institute of Neurological Disease and Stroke -Gaucher's Disease Information
Disabled World is an independent disability community established in 2004 to provide disability news and information to people with disabilities, seniors, their family and/or carers. See our homepage for informative news, reviews, sports, stories and how-tos. You can also connect with us on Twitter and Facebook or learn more about Disabled World on our about us page.
Disabled World provides general information only. The materials presented are never meant to substitute for professional medical care by a qualified practitioner, nor should they be construed as such. Financial support is derived from advertisements or referral programs, where indicated. Any 3rd party offering or advertising does not constitute an endorsement.
Cite This Page (APA): U.S. Food and Drug Administration. (2010, February 28). Gaucher Disease Therapy Approved by FDA. Disabled World. Retrieved August 19, 2022 from www.disabled-world.com/medical/pharmaceutical/gaucher-therapy.php
• Permalink: <a href="https://www.disabled-world.com/medical/pharmaceutical/gaucher-therapy.php">Gaucher Disease Therapy Approved by FDA</a>