August 25, 2009 / 12:50 PM / 8 years ago

UPDATE 2-Protalix's Gaucher drug wins fast-track review

* Aims to have data from a late-stage trial by October

* If approved, drug would compete with Genzyme’s Cerezyme

* Shares rise 1 percent. (Adds CEO comments, details on Gaucher drugs, closing shares)

By Ransdell Pierson

NEW YORK, Aug 25 (Reuters) - Protalix BioTherapeutics Inc (PLX.A) said on Tuesday U.S. regulators will review its experimental treatment for Gaucher disease on a fast-track basis, and it aims to have data from a late-stage trial of the drug by October.

The fast-track designation from the U.S. Food and Drug Administration means the agency will accept initial late-stage data instead of waiting for entire Phase III clinical trial data.

Experimental drugs seeking approval from the FDA are granted this designation when they are intended for treatment of a serious or life-threatening disease and demonstrate the potential to address unmet needs for such a condition.

If approved, the Protalix drug, called prGCD, would compete with Genzyme Corp’s GENZ.O Cerezyme, the only approved treatment. Genzyme ran into production problems earlier this year.

In patients with the rare Gaucher disease, organs such as the spleen and liver become enlarged due to the lack of an enzyme needed to break down fats.

Shares of Protalix rose 1 percent on the American Stock Exchange to $6.04. They jumped 30 percent on Aug. 17 after the tiny Israel-based biotechnology company said the FDA had approved its treatment protocol for prGCD.

This allows doctors to administer the drug as the Phase III trial of prGCD continues, before FDA approval, due to a continuing shortage of the Genzyme treatment.

David Aviezer, the chief executive of Protalix, said in an interview on Tuesday that many doctors around the world have asked his company to supply them its medicine, due to shortages of Cerezyme.

To qualify for prGCD, patients have to meet certain requirements, including willingness to become subjects of the ongoing Phase III trial of the medicine, Aviezer said.

Genzyme recently said it would shut down its plant in Boston after a virus halted production of Cerezyme and Fabrazyme, its treatment for Fabry disease, another rare condition.

Cerezyme is made in hamster ovary cells, while the Protalix drug is manufactured in cells taken from carrots, a difference that Aviezer said could confer safety advantages to his product.

“Plant cells cannot be affected by human or mammalian viruses, like incidents that happened with Genzyme,” he said. “We have a built-in biological firewall protecting against such situations.”

Aviezer said recent actions by the FDA, in approving the compassionate-use protocol and granting prGCD fast-track status, suggest the agency considers prGCD “a valid option.”

“We’re confident in the way the drug is moving ahead, and are confident in our ability to compete with Cerezyme,” he said. “We’re building up our credibility.” (Editing by John Wallace and Steve Orlofsky)

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