A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

Summary

Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system. This is the second trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD) which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.

Timeline

Start

2007-08

Primary completion

2009-09

Completion

2009-10

Outcome

Met primary endpoint

registry analysis (superiority or other test); ELELYSO 30 Units/kg; p <0.0001; one-sample t-test NCT00376168 ↗

registry analysis (superiority or other test); ELELYSO 60 Units/kg; p <0.0001; one-sample t-test NCT00376168 ↗

release “trial of UPLYSO (taliglucerase alfa, previously referred to as prGCD) in treatment naive patients diagnosed with Gaucher disease. The trial met its primary endpoint” protalix.com ↗

release “The pivotal Phase III clinical trial achieved its primary endpoint as ELELYSO significantly reduced spleen volume after nine months compared to baseline” protalix.com ↗

Publications

• Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.
• Zimran A, Brill-Almon E, Chertkoff R, Petakov M, Blanco-Favela F, Munoz ET, Solorio-Meza SE, Amato D, Duran G, Giona F, Heitner R, Rosenbaum H, Giraldo P, Mehta A, Park G, Phillips M, Elstein D, Altarescu G, Szleifer M, Hashmueli S, Aviezer D. Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood. 2011 Nov 24;118(22):5767-73. doi: 10.1182/blood-2011-07-366955. Epub 2011 Sep 6.
• Winckler T. [Enzyme replacement therapy for Gaucher's Disease]. Pharm Unserer Zeit. 2008;37(5):352-3. doi: 10.1002/pauz.200890067. No abstract available. German.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Taliglucerase alfa	Protein / enzyme biologic	30 iu/kg	Intravenous
Subject	Taliglucerase alfa	Protein / enzyme biologic	60 iu/kg	Intravenous

Indications