FDA aproves Elelyso (Pfizer) for Type 1 Gaucher Disease

The FDA on 1st May 2012 approved Elelyso (taliglucerase alfa) from Pfizer for long-term enzyme replacement therapy to treat a form of Gaucher Disease. 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. 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. The drug was developed by Protalix Biotherapeutics and is filed in the EU as Uplyso.