foodconsumer.org: FDA Approves Therapy to Treat Gaucher Disease

FDA Approves Therapy to Treat Gaucher Disease
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admin on 02/27/2010 12:48:00
 
The Food and Drug Administration (FDA) announced Feb 26 that it has approved
velaglucerase alfa for injection (VPRIV) to treat children and adults with a
rare genetic disorder called Gaucher disease.
Patients with Gaucher disease have deficiency of glucocerebrosidase, which cause
build-up of harmful amounts of a certain fatty substance (lipid) in the liver,
spleen, bones, bone marrow and nervous system, and make cells and organs
dysfunctional.
VPRIV manufactured by Shire Human Genetic Therapies Inc. of Cambridge, Mass is
indicated for treatment of Type 1 Gaucher disease as a long term enzyme
replacement therapy.  Currently, another drug known as Cerezyme or imiglucerase
is in short supply, the FDA says.
The safety and efficacy of VPRIV was assessed in three clinical stuides of 82
patients with Type 1 Gaucher disease aged 4 years and older.
Patients who previously used Cerezyme can be safely switched to VPRIV, said
Julie Beitz MD, of the FDA's office of Drug Evaluation II.
The adverse effects include allergic reactions, headache, dizziness, abdominal
pain, back pain, joint pain, nausea, fatigue/weakness, fever, and prolongation
of activated partial thromboplastin time, a measure of clotting time.
Patients with Type 1 Gaucher disease usually bruise easily and experience
fatigue due to anemia and low blood platelets. They also have an enlarged liver
and spleen, skeletal disorders, and, in some cases,lung and kidney impairment.
Gaucher disease symptoms can show up at any age although they usually are first
noticed in childhood or adolescence. The symptoms include easy bleeding and
bruising, excessive fatigue, anemia, weak bones, fracturing easily, bone and
joint pain, enlargement of the belly through increase in the volume of spleen
and liver.
By Jimmy Downs