Taliglucerase alfa is an intravenous enzyme replacement therapy approved for 
treatment of type 1 Gaucher disease (GD), and is the first available plant 
cell-expressed recombinant therapeutic protein. Herein, we report long-term 
safety and efficacy results of taliglucerase alfa in treatment-naïve adult 
patients with GD. Patients were randomized to receive taliglucerase alfa 30 or 
60 U/kg every other week, and 23 patients completed 36 months of treatment. 
Taliglucerase alfa (30 U/kg; 60 U/kg, respectively) resulted in mean decreases 
in spleen volume (50.1%; 64.6%) and liver volume (25.6%; 24.4%) with mean 
increases in hemoglobin concentration (16.0%; 35.8%) and platelet count (45.7%; 
114.0%), and mean decreases in chitotriosidase activity (71.5%; 82.2%). All 
treatment-related adverse events were mild to moderate in intensity and 
transient. The most common adverse events were nasopharyngitis, arthralgia, 
upper respiratory tract infection, headache, pain in extremity, and 
hypertension. These 36-month results of taliglucerase alfa in treatment-naïve 
adult patients with GD demonstrate continued improvement in disease parameters 
with no new safety concerns. These findings extend the taliglucerase alfa 
clinical safety and efficacy dataset. www.clinicaltrials.gov identifier 
NCT00705939. Am. J. Hematol. 91:656-660, 2016. © 2016 Wiley Periodicals, Inc.