Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease

Orphanet Journal of Rare Diseases
Ari ZimranG M Pastores

Abstract

Taliglucerase alfa is an enzyme replacement therapy (ERT) approved for treatment of adult and paediatric patients with Type 1 Gaucher disease (GD) in several countries and the first plant cell-expressed recombinant therapeutic protein approved by the US Food and Drug Administration for humans. Here, we review the findings across six key taliglucerase alfa clinical studies. A total of 33 treatment-naïve adult patients were randomized to taliglucerase alfa 30 U/kg or 60 U/kg in a 9-month, multicentre, randomized, double-blind, parallel-group, dose-comparison pivotal study, after which eligible patients continued into two consecutive extension studies; 17 treatment-naïve adult patients completed 5 total years of treatment with taliglucerase alfa. In the only ERT study focused on exclusively paediatric patients with GD, 11 treatment-naïve children were randomized to taliglucerase alfa 30 U/kg or 60 U/kg in a 12-month, multicentre, double-blind study; nine completed 3 total years of treatment in a dedicated paediatric extension study. The effect of switching patients from imiglucerase to taliglucerase alfa was also investigated in a separate 9-month study that included 26 adults and five children; 10 adults completed a total of 3 ye...Continue Reading

References

Dec 17, 2003·European Journal of Pediatrics·Gregory A GrabowskiAshok Vellodi
Jun 7, 2006·Archives of Pediatrics & Adolescent Medicine·Paige KaplanJohn D Yee
Dec 3, 2014·Blood Cells, Molecules & Diseases·Ari ZimranRaul Chertkoff

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Citations

Nov 18, 2018·Expert Review of Clinical Pharmacology·Punita Gupta, Gregory Pastores
Dec 14, 2018·Frontiers in Physiology·Valeriya V SolovyevaAlbert A Rizvanov
Jul 11, 2018·International Journal of Molecular Sciences·Uthailak NaphatsamonKazuhito Fujiyama
Jul 1, 2020·Expert Opinion on Biological Therapy·Saptashwa DattaHatem Zayed
Jan 10, 2021·Molecular Genetics and Metabolism·Gregory A GrabowskiPramod K Mistry

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Methods Mentioned

BETA
enzyme replacement therapy
glycosylation
X-ray
sedation

Clinical Trials Mentioned

NCT00376168
NCT00712348
NCT01132690
NCT01411228
NCT01422187

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