The US Food and Drug Administration (FDA) has awarded prGCD, a treatment for Gaucher disease produced by Protalix BioTherapeutics, orphan drug designation.
Gaucher disease is a lysosomal storage disorder resulting from 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.
Orphan drug designation is awarded by the FDA to drugs that may provide a significant therapeutic advantage over existing treatments.
Protalix president and CEO Dr David Aviezer said that the designation offers the company a number of benefits.
“The FDA’s orphan drug designation further strengthens our prGCD programme for treating Gaucher disease by offering important clinical development and commercialisation benefits,” Aviezer said.
prGCD is in a Phase III clinical trial which is scheduled to end in September, Protalix said.