Taliglucerase alfa (Protalix Biotherapeutics, Carmiel, Israel) is a novel plant 
cell-derived recombinant human β-glucocerebrosidase for Gaucher disease. A phase 
3, double-blind, randomized, parallel-group, comparison-dose (30 vs 60 U/kg body 
weight/infusion) multinational clinical trial was undertaken. Institutional 
review board approvals were received. A 9-month, 20-infusion trial used 
inclusion/exclusion criteria in treatment-naive adult patients with splenomegaly 
and thrombocytopenia. Safety end points were drug-related adverse events: Ab 
formation and hypersensitivity reactions. Primary efficacy end point was 
reduction in splenic volume measured by magnetic resonance imaging. Secondary 
end points were: changes in hemoglobin, hepatic volume, and platelet counts. 
Exploratory parameters included biomarkers and bone imaging. Twenty-nine 
patients (11 centers) completed the protocol. There were no serious adverse 
events; drug-related adverse events were mild/moderate and transient. Two 
patients (6%) developed non-neutralizing IgG Abs; 2 other patients (6%) 
developed hypersensitivity reactions. Statistically significant spleen reduction 
was achieved at 9 months: 26.9% (95% confidence interval [CI]: -31.9, -21.8) in 
the 30-unit dose group and 38.0% (95% CI: -43.4, -32.8) in the 60-unit dose 
group (both P < .0001); and in all secondary efficacy end point measures, except 
platelet counts at the lower dose. These results support safety and efficacy of 
taliglucerase alfa for Gaucher disease.