Spotlight on taliglucerase alfa in the treatment of pediatric patients with type 1 Gaucher disease

Figures & data

Table 1 Summary of phase III and IV taliglucerase alfa clinical trials, including an expanded access, open-label trial

Study	Identifier	Phase	Design	Patients	Taliglucerase dosing	End point
PB-06001	NCT00376168	III	9 month, double-blind, randomized, parallel-group, multicenter, dose-ranging, pivotal trial	Treatment-naive patients with GD	30 or 60 U/kg every 2 weeks	Changes from baseline in spleen and liver volume, platelet counts, hemoglobin level, and chitotriosidase activity
PB-06002	NCT00712348	III	9 month, open-label, switchover trial	Patients with stable GD treated with imiglucerase in previous 2 years	Dose equivalent to current imiglucerase every 2 weeks	Changes from baseline in spleen and liver volume, platelet counts, hemoglobin level, and chitotriosidase activity
PB-06003	NCT00705939	III	15 month, open-label, extension trial	Patients with GD, successfully completed PB-06-001	30 or 60 U/kg every 2 weeks	Changes from baseline in spleen and liver volume, platelet counts, hemoglobin level, and chitotriosidase activity
PB-06004	NCT00962260	NA	Expanded access, open-label trial	Adult patients with GD type 1, prior imiglucerase treatment discontinued or reduced due to shortage	Dose equivalent to current imiglucerase every 2 weeks	Safety
PB-06005	NCT01132690	IV	12 month, double-blind, multicenter, trial	Pediatric patients (2–18 years of age), ERT treatment-naïve	30 or 60 U/kg every 2 weeks	Primary: median percent changes in hemoglobin concentration from baseline Secondary: percent change in spleen and liver volume, platelet counts, and chitotriosidase activity
PB-06006	NCT01411228	III	24 month, double-blind, extension trial	Pediatric patients (2–18 years of age), who completed PB-06-002 or PB-06-005	30 or 60 U/kg every 2 weeks	Primary: median percent changes in hemoglobin concentration from baseline Secondary: percent change in spleen and liver volume, platelet counts, and chitotriosidase activity
PB-06007	NCT01422187	III	21 month, open-label, extension trial	Adult patients with GD, who completed PB-06-001 andPB-06003	30 or 60 U/kg every 2 weeks	Primary: median percent changes in hemoglobin concentration from baseline Secondary: percent change in spleen and liver volume, platelet counts, and chitotriosidase activity

Note: Reprinted from Molecular Genetics and Metabolism, vol 112. Grabowski GA, Golembo M, Shaaltiel Y. Taliglucerase alfa: an enzyme replacement therapy using plant cell expression technology. Pages: 1–8. Copyright 2014, with permission from Elsevier.¹⁸

Abbreviations: ERT, enzyme replacement therapy; GD, Gaucher disease; NA, not applicable.

Figure 1 PB-06-002 was a switchover study from imiglucerase to taliglucerase alfa.

Notes: ^aPatient had GD Type 3c and cardiac involvement requiring intervention due to decompensation. ^bPatients did not reach 33 total months of treatment due to early closure of study. Patients were either maintained on the same dose as received on imiglucerase, or if they were on <60 U/kg of imiglucerase, dose could be increased based on medical condition and at the investigator’s discretion. The five pediatric patients who completed this trial were enrolled in the PB-06-006 trial. Of these, one was lost to follow-up, two completed the 24 months duration of the extension trial, and two completed only 18 months of the extension trial due to early closure of the study; however, both patients continued to receive therapy through the compassionate use program. PB-06-005 involved the treatment of naïve pediatric patients who received either 30 or 60 U/kg of taliglucerase alfa for 12 months. Out of the 11 patients who completed the PB-06-005 trial, 10 were enrolled in the PB-06-006 extension trial. One patient had Type 3c GD and cardiac involvement requiring intervention due to decompensation and was not recruited into the extension phase, one patient dropped out from the trial, and nine patients completed the PB-06-006 extension trial. Out of the 15 patients enrolled in the PB-06006 extension trial, 11 completed. Reprinted from Blood Cells Mol Dis. Vol S1079–9796(16). Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, et al. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imiglucerase. Pages 30221–30222. Copyright 2016, with permission from Elsevier.²⁵
Abbreviation: GD, Gaucher disease.

GrabowskiGAGolemboMShaaltielYTaliglucerase alfa: an enzyme replacement therapy using pla nt cell expression technologyMol Genet Metab20141121824630271

 PubMed Web of Science ®Google Scholar

ZimranAGonzalez-RodriguezDEAbrahamovALong-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imigluceraseBlood Cells Mol Dis2016S10799796163022130222

 Google Scholar