DOI: 10.5061/dryad.75m46
title: Data from: Immunogenicity of glycans on biotherapeutic drugs produced in plant expression systems - the taliglucerase alfa story

description: Plants are a promising alternative for the production of biotherapeutics.
 Manufacturing in-planta adds plant specific glycans. To understand
 immunogenic potential of these glycans, we developed a validated method to
 detect plant specific glycan antibodies in human serum. Using this assay,
 low prevalence of pre-existing anti-plant glycan antibodies was found in
 healthy humans (13.5%) and in glucocerebrosidase-deficient Gaucher disease
 (GD) patients (5%). A low incidence (9% in naïve patient and none in
 treatment experienced patients) of induced anti-plant glycan antibodies
 was observed in GD patients after up to 30 months replacement therapy
 treatment with taliglucerase alfa, a version of human glucocerebrosidase
 produced in plant cells. Detailed evaluation of clinical safety and
 efficacy endpoints indicated that anti-plant glycan antibodies did not
 affect the safety or efficacy of taliglucerase alfa in patients. This
 study shows the benefit of using large scale human trials to evaluate the
 immunogenicity risk of plant derived glycans, and indicates no apparent
 risk related to anti-plant glycan antibodies.
description: Study Results -
 ClinicalTrials NCT00376168Results from Study
 PB-06-001, ‘A Phase III Multicenter, Randomized, Double-Blind Trial to
 Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell
 Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with
 Gaucher Disease’. ERT-naïve adult patients (N=32) with a total treatment
 duration of 9 months (study started on August 2007 and ended September
 2009). Results available at ClinicalTrials.gov: NCT00376168. Please
 acknowledge the National Library of Medicine (NLM) in subsequent use of
 this document. While using these results please cite the study in PLOS One
 and acknowledge the National Library of Medicine.Study Results -
 ClinicalTrials NCT00712348Results from Study
 PB-06-002, ‘A Phase 3 Multicenter, Open-label, Switchover Trial to Assess
 the Safety and Efficacy of Plant Cell Expressed Recombinant Human
 Glucocerebrosidase (Taliglucerase alfa) in Patients with GD Treated with
 Imiglucerase (Cerezyme®) Enzyme Replacement Therapy.’ Adult and pediatric
 patients (N=26 and 5, respectively) with a total treatment duration of 9
 months (study started on December 2008 and ended January 2013). Results
 available at ClinicalTrials.gov: NCT00712348. Please acknowledge the
 National Library of Medicine (NLM) in subsequent use of this document.
 While using these results please cite the study in PLOS One and
 acknowledge the National Library of Medicine.Study Results -
 ClinicalTrials NCT01132690Results for Study
 PB-06-005, ‘A Multicenter, Double-blind, Randomized Safety and Efficacy
 Study of Two Dose Levels of TGA in Pediatric patients with Gaucher
 Disease’. Pediatric patients (N=11) with a total treatment duration of 12
 months. (study started on August 2010 and ended July 2012). Results
 available at ClinicalTrials.gov: NCT01132690. Please acknowledge the
 National Library of Medicine (NLM) in subsequent use of this document.
 While using these results please cite the study in PLOS One and
 acknowledge the National Library of Medicine.