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Pfizer Announces FDA Approval Of Elelyso For Treatment Of Type 1 Gaucher Disease

Pfizer Inc.(PFE) and Protalix BioTherapeutics Inc. (PLX) said Tuesday that the U.S. Food and Drug Administration has approved ELELYSO for injection, an enzyme replacement therapy for the long-term treatment of adults with type 1 Gaucher disease.

According to the companies, ELELYSO (taliglucerase alfa) is the first FDA-approved plant cell-based enzyme replacement therapy, or ERT for Gaucher disease. It is also the first approved plant cell-expressed drug that is derived from ProCellEx, Protalix's proprietary manufacturing system, using genetically engineered carrot cells.

ELELYSO is a form of the human lysosomal enzyme glucocerebrosidase, used to treat Gaucher disease. The approval of ELELYSO is based on the review of Protalix's clinical development program.

In a study of 31 adult patients with Type 1 Gaucher disease naïve to enzyme replacement therapy, the safety and efficacy of ELELYSO was assessed. The trial was a nine-month, double blind, randomized study in patients with Gaucher disease-related enlarged spleens and thrombocytopenia.

Patients were randomized to receive ELELYSO at a dose of either 30 Units/kg or 60 Units/kg. Data showed the pivotal phase III clinical trial achieved its primary endpoint as ELELYSO significantly reduced spleen volume after nine months compared to baseline in both treatment groups.

Twenty-six patients continued to be treated with ELELYSO in an extension of this study in a blinded manner for a total treatment duration of 24 months. The data demonstrated continued improvement in the clinical parameters.

The most common adverse reactions during clinical studies were infusion reactions. Other commonly observed adverse reactions in less than ten percent of patients were nasopharyngitis, throat infection, headache, arthralgia, influenza, pyelonephritis, back pain and extremity pain.

Gaucher disease is an inherited lysosomal storage disorder in humans that affects an estimated 10,000 people worldwide and can cause severe and debilitating symptoms, including: enlargement of the liver and spleen, various forms of bone disease, easy bruising, and anemia.

In November, 2009, Pfizer and Protalix entered into an agreement to develop and commercialize taliglucerase alfa. Under the terms, Protalix retained exclusive commercialization rights in Israel, while Pfizer received exclusive licensing rights for the commercialization of ELELYSO in all other markets. As part of this agreement, and at the conclusion of the FDA approval process, Protalix will transfer the ELELYSO NDA and IND to Pfizer.

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