Protalix gets FDA orphan drug status for Gaucher's treatment

The company plans filing a New Drug Application (NDA) with the FDA before the end of 2009.

Protalix Biotherapeutics Inc. (AMEX:PLX) has obtained orphan drug designation from the US Food and Drug Administration (FDA) for prGCD, the company’s proprietary plant cell-derived treatment of Gaucher disease. prGCD is a plant cell expressed recombinant form of glucocerebrosidase (GCD).

Gaucher disease caused by 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.

Protalix president and CEO Dr. David Aviezer said, “The FDA's orphan drug designation further strengthens our prGCD program for treating Gaucher disease by offering important clinical development and commercialization benefits."

prGCD is undergoing a Phase III clinical trial, which is scheduled to end this month. Protalix plans to announce initial results in October 2009, and file a New Drug Application (NDA) with the FDA before the end of the year. Following FDA approval of the company’s treatment protocol for the drug, Protalix expects to start treating patients worldwide for Gaucher's disease.

Orphan drug designation applies 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 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.

Protalix's share rose 3.7% at the opening on the American Stock Exchange today to $7.65, giving a market cap of $585 million.

Published by Globes [online], Israel business news - - on September 8, 2009

© Copyright of Globes Publisher Itonut (1983) Ltd. 2009

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