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Protalix BioTherapeutics, Inc. (PLX) Complex Receives FDA’s Orphan Drug Designation

Earlier today, Protalix BioTherapeutics, Inc. announced exciting news regarding its proprietary plant cell, prGCD. The company has received notice from the U.S. Food and Drug Administration that the Office of Orphan Products Development has granted orphan drug designation to prGCD, which contains an expressed recombinant form of glucocerebrosidase (GCD), for the treatment of Gaucher disease. Gaucher disease is a lysosomal storage disorder resulting from a deficiency or mutation of the GCD enzyme. The disease affects the liver, spleen, bone marrow, and in some cases, the central nervous system.

Orphan drug designation is granted to drugs that may potentially provide a significant therapeutic advantage over existing treatments. The chosen drugs often target conditions affecting 200,000 or fewer US patients per year. Orphan drug status grants a faster review time of the drug’s New Drug Application, and qualifies the drug for possible funding and tax savings to support clinical trials, as well as other financial incentives. Additionally, the sponsor company must meet certain conditions established by the FDA to remain eligible for granting of the orphan drug status.

prGCD is currently being evaluated for the treatment of Gaucher disease in a Phase III clinical trial, scheduled to be completed by the end of September. The company plans to announce top-line results from the Phase III trial in October 2009, and to complete filing of an NDA with the FDA before the end of the year.

“The FDA’s orphan drug designation further strengthens our prGCD program for treating Gaucher disease by offering important clinical development and commercialization benefits,” said Dr. David Aviezer, the Company’s President and Chief Executive Officer.

Following the FDA’s recent approval of Protalix’s treatment protocol, the company filed applications for medical Institutional Review Board approvals in hospitals worldwide.

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