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Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 18.

Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.

Author information

1
National Centre for Inherited Metabolic Disorders, Mater Misericordiae University Hospital, Dublin, Ireland.
2
Department of Genetics, Emory University School of Medicine, Atlanta, Georgia.
3
Department of Ophthalmology, Emory University School of Medicine, Atlanta, Georgia.
4
Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Diseases, Belgrade University Medical School, Belgrade, Serbia.
5
CIBERER, Hospital Universitario Miguel Servet, Zaragoza, Spain.
6
Rambam Medical Center, Haifa, Israel.
7
Mount Sinai Hospital, Toronto, Ontario, Canada.
8
Royal Melbourne Hospital, Victoria, Australia.
9
Protalix BioTherapeutics, Carmiel, Israel.
10
Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel.

Abstract

Taliglucerase alfa is the first available plant cell-expressed human recombinant therapeutic protein. It is indicated for treatment of patients with type 1 Gaucher disease (GD) in adult and pediatric patients in several countries. Study PB-06-002 examined the safety and efficacy of taliglucerase alfa for 9 months in patients who previously received imiglucerase. The results of adult patients from Study PB-06-002 who continued receiving taliglucerase alfa in extension Study PB-06-003 for up to 36 months are reported here. Eighteen patients received at least one dose of taliglucerase alfa in Study PB-06-003; 10 patients completed 36 total months of therapy, and four patients who transitioned to commercial drug completed 30-33 months of treatment. In patients who completed 36 total months of treatment, mean percent (±standard error) changes from baseline/time of switch to taliglucerase alfa to 36 months were as follows: hemoglobin concentration, -1.0% (±1.9%; n = 10); platelet count, +9.3% (±9.8%; n = 10); spleen volume measured in multiples of normal (MN), -19.8% (±9.9%; n = 7); liver volume measured in MN, +0.9% (±5.4%; n = 8); chitotriosidase activity, -51.5% (±8.1%; n = 10); and CCL18 concentration, -36.5 (±8.0%; n = 10). Four patients developed antidrug antibodies, including one with evidence of neutralizing activity in vitro. All treatment-related adverse events were mild or moderate and transient. The 36-month results of switching from imiglucerase to taliglucerase alfa treatment in adults with GD provide further data on the clinical safety and efficacy of taliglucerase alfa beyond the initial 9 months of the original study. www.clinicaltrials.gov identifier NCT00705939. Am. J. Hematol. 91:661-665, 2016.

PMID:
27102949
PMCID:
PMC5084808
DOI:
10.1002/ajh.24399
[Indexed for MEDLINE]
Free PMC Article

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