Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease
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 huma...
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doaj-18c97debc4bc49979375369bd515e8b92020-11-24T23:50:59ZengBMCOrphanet Journal of Rare Diseases1750-11722018-02-0113111010.1186/s13023-018-0776-8Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher diseaseAri Zimran0Michael Wajnrajch1Betina Hernandez2Gregory M. Pastores3Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical SchoolPfizer IncPfizer IncUniversity College Dublin and the National Centre for Inherited Metabolic Disorders, Mater Misericordiae University HospitalAbstract 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 years and two children completed a total of 2.75 years of taliglucerase alfa treatment in the extension studies. All studies evaluated safety and spleen volume, liver volume, platelet count, haemoglobin concentration, and biomarkers as measures of efficacy. Detailed results from baseline through the end of these studies are presented. Taliglucerase alfa was well tolerated, and adverse events were generally mild/moderate in severity and transient. Treatment with taliglucerase alfa resulted in improvements (treatment-naïve patients) or stability (patients switched from imiglucerase) in visceral, haematologic, and biomarker parameters. Together, this comprehensive data set supports the treatment of adult and paediatric patients with GD who are naïve to ERT or who have previously been treated with imiglucerase.http://link.springer.com/article/10.1186/s13023-018-0776-8AnaemiaEnzyme replacement therapyGaucher diseaseImigluceraseHepatomegalyPaediatrics |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Ari Zimran Michael Wajnrajch Betina Hernandez Gregory M. Pastores |
spellingShingle |
Ari Zimran Michael Wajnrajch Betina Hernandez Gregory M. Pastores Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease Orphanet Journal of Rare Diseases Anaemia Enzyme replacement therapy Gaucher disease Imiglucerase Hepatomegaly Paediatrics |
author_facet |
Ari Zimran Michael Wajnrajch Betina Hernandez Gregory M. Pastores |
author_sort |
Ari Zimran |
title |
Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease |
title_short |
Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease |
title_full |
Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease |
title_fullStr |
Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease |
title_full_unstemmed |
Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease |
title_sort |
taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with gaucher disease |
publisher |
BMC |
series |
Orphanet Journal of Rare Diseases |
issn |
1750-1172 |
publishDate |
2018-02-01 |
description |
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 years and two children completed a total of 2.75 years of taliglucerase alfa treatment in the extension studies. All studies evaluated safety and spleen volume, liver volume, platelet count, haemoglobin concentration, and biomarkers as measures of efficacy. Detailed results from baseline through the end of these studies are presented. Taliglucerase alfa was well tolerated, and adverse events were generally mild/moderate in severity and transient. Treatment with taliglucerase alfa resulted in improvements (treatment-naïve patients) or stability (patients switched from imiglucerase) in visceral, haematologic, and biomarker parameters. Together, this comprehensive data set supports the treatment of adult and paediatric patients with GD who are naïve to ERT or who have previously been treated with imiglucerase. |
topic |
Anaemia Enzyme replacement therapy Gaucher disease Imiglucerase Hepatomegaly Paediatrics |
url |
http://link.springer.com/article/10.1186/s13023-018-0776-8 |
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