Elelyso® for Gaucher Disease

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Elelyso® for Gaucher Disease


Gaucher disease, also known as glucocerebrosidase deficiency, is an autosomal recessive disorder and the most prevalent Lysosomal Storage Disorder (LSD) in the world. It is one of a group of disorders that affect specific enzymes that normally break down materials for reuse in the cells. If the enzymes are missing or do not work properly, the materials can build up and become toxic. Gaucher disease occurs when a lipid called glucosylceramide accumulates in the bone marrow, lungs, spleen, liver, and sometimes the brain. It is present in approximately 1 in 20,000 live births. Gaucher disease symptoms can include fatigue, anemia, easy bruising and bleeding, severe bone pain and easily broken bones, and distended stomach due to an enlarged spleen and thrombocytopenia. The gold standard of care for Gaucher disease is enzyme replacement therapy (ERT). Enzyme replacement therapy is a medical treatment in which recombinant enzymes are injected into patients in whom the enzyme is lacking or dysfunctional. For Gaucher disease, recombinant glucocerebrosidase (GCD) is injected to replace the mutated or deficient natural GCD enzyme. ERT treatment may help people live normal lives.



On May 1, 2012, the U.S. Food and Drug Administration (FDA) approved taliglucerase alfa for injection, as an enzyme replacement therapy (ERT) for the long-term treatment of adult patients with a confirmed diagnosis of type 1 Gaucher disease. Taliglucerase alfa is a proprietary, recombinant form of glucocerebrosidase (GCD) is the company’s first approved drug product developed using ProCellEx®. Taliglucerase alfa was also approved by the Israeli Ministry of Health in September 2012, by the Brazilian Ministry of Health in March 2013, and by the applicable regulatory authorities of certain other countries. Taliglucerase alfa is the first plant cell-based recombinant therapeutic protein approved by the FDA or any other major regulatory authority. In 2009 Protalix entered into a collaboration agreement with Pfizer Inc. for the continued development and commercialization of Elelyso®. In October 2015, this agreement was amended providing Pfizer worldwide marketing rights for Elelyso® , marketed in Latin America under the name Uplyso™) with exception of Brazil. In Brazil, taliglucerase alfa is sold under the brand name Bio-Manguinhos alfataliglicerase under a special supply and technology transfer agreement with Fundação Oswaldo Cruz.


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