Israel: Protalix gets FDA orphan drug status for Gaucher's treatment

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."
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Anti Boycott Israel blog: maladies orphelines, maladie de Gaucher