Elelyso Approved for Gaucher Disease
People with rare disorder lack enzyme
TUESDAY, May 1, 2012 (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.
Medline Plus has more about Gaucher disease.