FDA Grants Protalix Orphan Drug Designation for prGCD

FDA Grants Protalix Orphan Drug Designation for prGCD

CARMIEL, Israel, September 8, 2009 (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, 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

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

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.

Safe Harbor Statement:

To the extent that statements in this press release are not strictly historical, all such

statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private

Securities Litigation Reform Act of 1995. These forward-looking statements are subject to known

and unknown risks and uncertainties that may cause actual future experience and results to differ

materially from the statements made. These statements are based on our current beliefs and

expectations as to such future outcomes. Drug discovery and development involve a high degree of

risk. Factors that might cause material differences include, among others, risks relating to: the

successful preclinical development of our product candidates; the completion of clinical trials;

the review process of the FDA, foreign regulatory bodies and other governmental regulation,

including the FDA s review of any filings we make in connection with the phase III trial; delays in

the FDA s or other health regulatory authorities approval of any applications we file or refusals

to approve such filings; prGCD s continuing to be eligible for expedited review under the fast

track and orphan drug programs, if we file an NDA for prGCD; refusals by such regulatory

authorities to approve the marketing and sale of a drug product even after acceptance of an

application we file for any such drug product; the identification of lead compounds; the risk that

we may fail to satisfy certain conditions relating to grants we have received from the Office of

the Chief Scientist of Israel s Ministry of Industry and Trade which may lead to our being required

to refund grants previously received

together with interest and penalties; the risk that the Office of the Chief Scientist may not

deliver to us all of the funds awarded to us; uncertainties related to the ability to attract and

retain partners for our technologies and products under development; and other factors described in

our filings with the Securities and Exchange Commission. Under our approved treatment protocol,

prGCD might be provided only to a limited number of patients and only for a limited time, and the

medical institutional review board of hospitals may not choose to start treating patients under our

extended access treatment protocol in a timely manner, if at all. Pharmaceutical and biotechnology

companies have suffered significant setbacks in advanced clinical trials, even after promising

results in earlier clinical trials or in preliminary findings for such clinical trials. The FDA s

approval of the treatment protocol for prGCD, the fast track approval, or the orphan

drug designation will not have any effect on the FDA s approval of any NDA we file with respect to

prGCD, if any, and the review by the FDA of any data from the Phase III clinical development

programs in connection with the approval of the treatment protocol will not have any effect on the

FDA s subsequent review of our complete Phase III clinical trial data in the future. The

statements are valid only as of the date hereof and we disclaim any obligation to update this

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