Yesterday, Protalix BioTherapeutics, Inc. (PLX) announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to prGCD for the treatment of Gaucher disease, an enzyme disorder that can cause liver and neurological problems.

The drug is currently being evaluated in a phase III trial which is scheduled to end this month. Top-line results are expected in October 2009. The company intends to file a New Drug Application (NDA) with the FDA before the end of the year.

Drugs that treat diseases affecting 200,000 or fewer U.S. patients per year are granted “orphan drug” status by the FDA. Drugs in this umbrella qualify for priority and potentially faster review times. They also enjoy funding and tax savings to support its clinical development.

Last month, the U.S. agency granted Fast-Track Designation to the drug. This process allows the filing of different sections of the NDA as and when available instead of filing all the sections at once. Furthermore, the status aims at filling an unmet medical need to make important new treatments available to patients sooner than expected.

The only enzyme replacement therapy currently approved for Gaucher is Genzyme’s (GENZ) Cerezyme. However, Genzyme temporarily stopped production and shipments of Cerezyme to remove viral contamination at one of its plants. Although Genzyme has resumed production, new shipments of Cerezyme will not be available until later this year.

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