Genzyme

FDA Approves Protalix, Pfizer Drug To Treat Gaucher Disease

The U.

S. Food and Drug Administration on Tuesday approved a new treatment developed by Protalix BioTherapeutics Inc. (PLX, PLX.TV) for adults with a type of Gaucher disease, a rare genetic disorder.The treatment, taliglucerase alfa, will be sold in the U.S. under the brand name Elelyso by Pfizer Inc. (PFE).Pfizer also will sell the drug in most places outside the U.S. The drug, which is the first plant-derived cell-based enzyme replacement, replaces an enzyme called glucocerebrosidase in people with type 1 Gaucher disease. Elelyso is produced using genetically engineered carrot cells.People with Gaucher disease lack glucocerebrosidase, which causes an accumulation of a fatty substance inside cells. The fatty accumulation can lead to enlargement and malfunctioning of the liver, spleen, bone marrow and occasionally, the lung, kidney, and intestine. About 6,000 people in the U.S. have type 1 Gaucher disease, according to the FDA.Pfizer and Protalix have already been making Elelyso availab...

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