A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase.

Abstract

Taliglucerase alfa is a β-glucosidase enzyme replacement therapy (ERT) approved in the US and other countries for the treatment of Gaucher disease (GD) in adults and is approved in pediatric and adult patients in Australia and Canada. It is the first approved plant cell-expressed recombinant human protein. A Phase 3, multicenter, open-label, 9-month study assessed safety and efficacy of switching to taliglucerase alfa in adult and pediatric patients with GD treated with imiglucerase for at least the previous 2years. Patients with stable disease were offered taliglucerase alfa treatment using the same dose (9-60U/kg body weight) and regimen of administration (every 2weeks) as imiglucerase. This report summarizes results from 26 adult and 5 pediatric patients who participated in the trial. Disease parameters (spleen and liver volumes, hemoglobin concentration, platelet count, and biomarker levels) remained stable through 9months of treatment in adults and children following the switch from imiglucerase. All treatment-related adverse events were mild or moderate in severity and transient in nature. Exploratory parameters of linear growth and development showed positive outcomes in pediatric patients. These findings provide evidence of the efficacy and safety profile of taliglucerase alfa as an ERT for GD in patients previously treated with imiglucerase. This trial was registered at www.clinicaltrials.gov as # NCT00712348.

• Authors: Gregory M Pastores, Milan Petakov, Pilar Giraldo, Hanna Rosenbaum, Jeffrey Szer, Patrick B Deegan, Dominick J Amato, Eugen Mengel, Ee Shien Tan, Raul Chertkoff, Einat Brill-Almon, Ari Zimran
• Journal: Blood cells, molecules & diseases (Blood Cells Mol Dis) Vol. 53 Issue 4 Pg. 253-60 (Dec 2014) ISSN: 1096-0961 [Electronic] United States
• PMID: 24950666 (Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2014 Elsevier Inc. All rights reserved.

Chemical References

• Hemoglobins
• Recombinant Proteins
• beta-Glucosidase
• Glucosylceramidase
• taliglucerase alfa
• imiglucerase

Topics

• Adolescent
• Adult
• Aged
• Blood Platelets (drug effects)
• Child
• Drug Substitution
• Enzyme Replacement Therapy
• Gaucher Disease (drug therapy, enzymology, genetics, pathology)
• Gene Expression
• Glucosylceramidase (biosynthesis, therapeutic use)
• Hemoglobins (metabolism)
• Humans
• Liver (drug effects, enzymology, pathology)
• Male
• Platelet Count
• Recombinant Proteins (biosynthesis, therapeutic use)
• Spleen (drug effects, enzymology, pathology)
• Young Adult
• beta-Glucosidase (deficiency, genetics)