FDA Grants Orphan Status to Protalix's Gaucher Disease Treatment

Protalix BioTherapeutics, Inc. (Carmiel, Israel) gained orphan drug status from the US FDA for prGCD, a development drug for Gaucher’s disease, on September 9. The orphan drug designation for prGCD was granted by the FDA’s Office of Orphan Products Development and comes less than a month after the drug received fast-track designation from the FDA.
 
The orphan drug designation is another step to help Gaucher’s patients in the wake of the current shortage of Cerezyme, the only drug currently approved for the disease. The Cerezyme shortage occurred because the manufacturer, Genzyme, halted production in mid-June because of virus contamination at its plant in Allston Landing, MA.
 
The FDA grants orphan drug designation to drugs that may provide a significant therapeutic advantage over existing treatments and target conditions affecting 200,000 or fewer US patients per year. The status allows a faster review time for the drug’s new drug application (NDA) and qualifies the drug for possible funding and tax savings.
 
The Phase 3 trial of prGCD for the treatment of Gaucher disease is scheduled to end this month and Protalix anticipates reporting top-line results next month. The company plans to complete filing of the prGCD NDA before the end of the year.
 
Earlier this week, Shire plc also completed its NDA submission for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease.
 
Previous coverage:
 
Shire Completes NDA for Gaucher's Disease Treatment
 
Gaucher Patients Can Be Treated with Protalix's Drug During Cerezyme Shortage 
Protalix's Gaucher Drug May Fill Gap Left from Genzyme Plant Closure 
Genzyme Detects Virus Contamination of Bioreactor, Halts Production