Elelyso Approved for Gaucher Disease05/01/12
TUESDAY, May 1 (HealthDay News) -- Elelyso (taliglucerase alfa)
has been approved by the U.S. Food and Drug Administration as a
long-term enzyme replacement therapy for people with a rare genetic
disorder called type 1 Gaucher disease.
People with the disorder don't produce enough of an enzyme
called glucocerebrosidase. This causes the buildup of fatty lipids
in the spleen, liver, kidneys and other organs. Warning signs of
the disorder may include anemia, low blood platelets and bone
problems, the FDA said in a news release. About 6,000 people in the
United States are thought to have the disorder.
Elelyso, an every-other-week professionally administered
injection, was clinically evaluated in a study of 56 people with
type 1 Gaucher disease. The most common side effects included
allergic reactions, headache, chest pain, fatigue, increased blood
pressure, back or joint pain, and flushing.
Elelyso is produced and distributed by New York City-based
Pfizer, under license from Protalix BioTherapeutics.
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