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.
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
Mastering Antibody-Drug Conjugates
December 19th 2024In this episode, we explore BIOVECTRA’s capabilities in antibody-drug conjugate (ADC) manufacturing, from complex conjugation chemistry to synthesis of highly potent payloads. We’ll also showcase how BIOVECTRA’s extensive experience in complex chemistries and specialized small molecule manufacturing gives them a unique perspective, strengthening their approach to ADC production and ensuring clients receive custom solutions across all project stages.
Drug Shortages and Complying with FDA’s 21 CFR 211.110 Guidance
April 2nd 2025Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, and Rona LeBlanc-Rivera, PhD, principal consultant, Regulatory Affairs at Regulatory Compliance Associates, answer some questions about FDA’s January 2025 21 CFR 211.110 guidance document.
