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Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease.
- Source :
-
Blood [Blood] 2011 Nov 24; Vol. 118 (22), pp. 5767-73. Date of Electronic Publication: 2011 Sep 06. - Publication Year :
- 2011
-
Abstract
- Taliglucerase alfa (Protalix Biotherapeutics, Carmiel, Israel) is a novel plant cell-derived recombinant human β-glucocerebrosidase for Gaucher disease. A phase 3, double-blind, randomized, parallel-group, comparison-dose (30 vs 60 U/kg body weight/infusion) multinational clinical trial was undertaken. Institutional review board approvals were received. A 9-month, 20-infusion trial used inclusion/exclusion criteria in treatment-naive adult patients with splenomegaly and thrombocytopenia. Safety end points were drug-related adverse events: Ab formation and hypersensitivity reactions. Primary efficacy end point was reduction in splenic volume measured by magnetic resonance imaging. Secondary end points were: changes in hemoglobin, hepatic volume, and platelet counts. Exploratory parameters included biomarkers and bone imaging. Twenty-nine patients (11 centers) completed the protocol. There were no serious adverse events; drug-related adverse events were mild/moderate and transient. Two patients (6%) developed non-neutralizing IgG Abs; 2 other patients (6%) developed hypersensitivity reactions. Statistically significant spleen reduction was achieved at 9 months: 26.9% (95% confidence interval [CI]: -31.9, -21.8) in the 30-unit dose group and 38.0% (95% CI: -43.4, -32.8) in the 60-unit dose group (both P < .0001); and in all secondary efficacy end point measures, except platelet counts at the lower dose. These results support safety and efficacy of taliglucerase alfa for Gaucher disease.
- Subjects :
- Adult
Aged
Algorithms
Double-Blind Method
Female
Gene Expression Regulation, Enzymologic
Gene Expression Regulation, Plant
Glucosylceramidase genetics
Glucosylceramidase metabolism
Humans
Male
Middle Aged
Placebos
Plant Cells enzymology
Recombinant Proteins genetics
Recombinant Proteins metabolism
Transfection
Treatment Outcome
Young Adult
Enzyme Replacement Therapy methods
Gaucher Disease drug therapy
Glucosylceramidase therapeutic use
Plant Cells metabolism
Subjects
Details
- Language :
- English
- ISSN :
- 1528-0020
- Volume :
- 118
- Issue :
- 22
- Database :
- MEDLINE
- Journal :
- Blood
- Publication Type :
- Academic Journal
- Accession number :
- 21900191
- Full Text :
- https://doi.org/10.1182/blood-2011-07-366955