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Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease.
Orphanet J Rare Dis. 2018 02 23; 13(1):36.OJ

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.

Authors+Show Affiliations

Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School, 12 Bayit Street, P.O. Box 3235, 91031, Jerusalem, Israel. azimran@gmail.com.Pfizer Inc, New York, NY, USA.Pfizer Inc, New York, NY, USA.University College Dublin and the National Centre for Inherited Metabolic Disorders, Mater Misericordiae University Hospital, Dublin, Ireland.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't
Review

Language

eng

PubMed ID

29471850

Citation

Zimran, Ari, et al. "Taliglucerase Alfa: Safety and Efficacy Across 6 Clinical Studies in Adults and Children With Gaucher Disease." Orphanet Journal of Rare Diseases, vol. 13, no. 1, 2018, p. 36.
Zimran A, Wajnrajch M, Hernandez B, et al. Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease. Orphanet J Rare Dis. 2018;13(1):36.
Zimran, A., Wajnrajch, M., Hernandez, B., & Pastores, G. M. (2018). Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease. Orphanet Journal of Rare Diseases, 13(1), 36. https://doi.org/10.1186/s13023-018-0776-8
Zimran A, et al. Taliglucerase Alfa: Safety and Efficacy Across 6 Clinical Studies in Adults and Children With Gaucher Disease. Orphanet J Rare Dis. 2018 02 23;13(1):36. PubMed PMID: 29471850.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease. AU - Zimran,Ari, AU - Wajnrajch,Michael, AU - Hernandez,Betina, AU - Pastores,Gregory M, Y1 - 2018/02/23/ PY - 2017/11/29/received PY - 2018/02/15/accepted PY - 2018/2/24/entrez PY - 2018/2/24/pubmed PY - 2019/3/15/medline KW - Anaemia KW - Enzyme replacement therapy KW - Gaucher disease KW - Hepatomegaly KW - Imiglucerase KW - Paediatrics KW - Splenomegaly KW - Taliglucerase alfa SP - 36 EP - 36 JF - Orphanet journal of rare diseases JO - Orphanet J Rare Dis VL - 13 IS - 1 N2 - 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. SN - 1750-1172 UR - https://www.unboundmedicine.com/medline/citation/29471850/Taliglucerase_alfa:_safety_and_efficacy_across_6_clinical_studies_in_adults_and_children_with_Gaucher_disease_ L2 - https://ojrd.biomedcentral.com/articles/10.1186/s13023-018-0776-8 DB - PRIME DP - Unbound Medicine ER -