FDA Grants Protalix Orphan Drug Designation for prGCD

08 Sept 2009 - CARMIEL, Israel--(BUSINESS WIRE)--Protalix BioTherapeutics, Inc. (NYSE-Amex:PLX), announced today that it has received notice from the U.S. Food and Drug Administration (FDA) that the FDA’s Office of Orphan Products Development has granted orphan drug designation to prGCD, the Company’s proprietary plant cell 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 that can cause damage to the liver, spleen, bone marrow and in some cases, the central nervous system.

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

“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.

prGCD is currently being evaluated for the treatment of Gaucher disease in a Phase III clinical trial which is scheduled to end this month. 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. In addition, after the FDA’s recent approval of the Company’s treatment protocol for prGCD, the Company filed applications for medical Institutional Review Board (IRB) approvals in hospitals worldwide and expects to start treating patients under the extended access treatment protocol.

About Protalix BioTherapeutics
Protalix is a biopharmaceutical company. Its goal is to become a fully integrated biopharmaceutical company focused on the development and commercialization of proprietary recombinant therapeutic proteins to be expressed through its proprietary plant cell based expression system. Protalix’s ProCellEx(TM) presents a proprietary method for the expression of recombinant proteins that Protalix believes will allow for the cost-effective, industrial-scale production of recombinant therapeutic proteins in an environment free of mammalian components and viruses. Protalix is conducting a Phase III pivotal study for its lead product candidate, prGCD, to be used in enzyme replacement therapy for Gaucher disease, a rare and serious lysosomal storage disorder in humans with severe and debilitating symptoms. Protalix and the U.S. Food and Drug Administration (FDA) agreed on the final design of the pivotal Phase III clinical trial through the FDA’s Special Protocol Assessment (SPA) process. In addition, Protalix has received Fast Track Designation from the FDA for prGCD and the FDA’s Office of Orphan Products Development has granted orphan drug designation to prGCD. Protalix is also advancing additional recombinant biopharmaceutical drug development programs.