Protalix Receives Fast Track Designation for Gaucher's Drug

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Protalix BioTherapeutics received "fast track" designation from the US FDA yesterday for prGCD, a development drug for Gaucher's disease.

Protalix BioTherapeutics (Carmiel, Israel) received "fast track" designation from the US Food and Drug Administration yesterday for prGCD, a development drug for Gaucher’s disease. The designation is the latest step to help Gaucher's patients in the wake of the current shortage of Cerezyme, the only drug currently approved for the disease.

Fast track designation expedites the review of drugs to treat serious diseases and fill an unmet medical need. The process allows the company to file sections of the new drug application (NDA) as they become available instead of filing all sections at once. The agency will begin its review and proceed on a rolling basis as sections are completed and submitted for review.

Last week, the company received FDA approval for a treatment protocol for prGCD, which allows physicians to treat patients while its pivotal Phase 3 clinical trial is still ongoing. Protalix expects to complete the Phase 3 trial in September, to report top-line results in October, and to complete the NDA filing before the end of the year.

The Cerezyme shortage occurred because the drug's manufacturer, Genzyme (Cambridge, MA), halted production of Cerezyme in mid-June because of virus contamination in a bioreactor at its plant in Allston Landing, MA. Genzyme has resumed manufacturing at the Allston plant following sanitization, but the Cerezyme shortage is expected to continue through the end of the year.