A pharmacokinetic analysis of a novel enzyme replacement therapy with Gene-Activated® human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease

Zimran, Ari; Loveday, Kenneth S.; Fratazzi, Candida; Elstein, Deborah

doi:10.1016/j.bcmd.2007.02.008

Cited by 56 publications

(36 citation statements)

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“…Velaglucerase alfa pharmacokinetic profile (data not shown) was consistent with that observed in the Phase 1/2 study [16]. Safety Fifteen (60%; n56 for 60 U/kg group; n59 for 45 U/kg group) of the 25 participants experienced at least one AE considered by the investigator to be related to velaglucerase alfa: 14 of 15 of these were considered to be infusionrelated.…”

Section: Efficacysupporting

confidence: 68%

Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study

et al. 2013

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Type 1 Gaucher disease (GD1), resulting from glucocerebrosidase deficiency, leads to splenomegaly, hepatomegaly, anemia, thrombocytopenia, and bone involvement. Current standard treatment is enzyme replacement therapy. Velaglucerase alfa is an enzyme replacement product for GD1, with the same amino acid sequence as naturally occurring human glucocerebrosidase. This multinational, Phase 3 trial evaluated the efficacy and safety of two doses of velaglucerase alfa in 25 treatment‐naïve, anemic patients with GD1 (4–62 years of age), randomized to intravenous velaglucerase alfa 60 U/kg (n=12) or 45 U/kg body weight (n=13) every other week for 12 months. The primary endpoint was change from baseline in hemoglobin concentration in the 60 U/kg arm. At 12 months, mean hemoglobin concentrations increased from baseline [60 U/kg: +23.3%; +2.43 g/dL (P<0.001); 45 U/kg: +23.8%; +2.44 g/dL (P<0.001)], as did mean platelet counts [60 U/kg: +65.9%; +50.9 × 109/L (P=0.002); 45 U/kg: +66.4%; +40.9 × 109/L(P=0.01)]. Mean splenic volume decreased from baseline [60 U/kg: −50.4%, from 14.0 to 5.8 multiples of normal (MN) (P=0.003); 45 U/kg: −39.9%, from 14.5 to 9.5 MN (P=0.009)]. No drug‐related serious adverse events or withdrawals were observed. One patient developed antibodies. Velaglucerase alfa was generally well tolerated and effective for adults and children with GD1 in this study. All disease‐specific parameters measured demonstrated clinically meaningful improvements after 12 months. Am. J. Hematol. 88:166–171, 2013. © 2012 Wiley Periodicals, Inc.

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Section: Efficacysupporting

confidence: 68%

Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study

et al. 2013

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“…[1][2][3][4] Nonetheless, the small sample size is a limitation of the study. Safety is also comparable with that of velaglucerase alfa (VPRIV; Shire HGT), manufactured in a human fibroblast cell line 19,20 that was in clinical trials at approximately the same time as taliglucerase alfa, and like taliglucerase alfa, was allowed by regulatory agencies (FDA, European Medicines Agency [EMA]), and the Israeli Ministry of Health) in Early Access Programs as a stop-gap measure for patients requiring ERT during the global imiglucerase shortage. 10,11 Velaglucerase alfa was approved in many countries in 2010.…”

Section: Discussionmentioning

confidence: 90%

Pivotal trial with plant cell–expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease

Zimran

Brill‐Almon

Chertkoff

et al. 2011

Blood

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“…Three ERTs are available for the treatment of Type 1 GD: imiglucerase, produced in a Chinese hamster ovary cell culture system [8,9]; velaglucerase alfa, produced in a human fibroblast cell system [10,11]; and taliglucerase alfa, an ERT produced in a plant cell-based expression system [12,13]. Taliglucerase alfa is the first US Food and Drug Administration-approved plant cellexpressed recombinant therapeutic protein [13,14].…”

Section: Introductionmentioning

confidence: 99%

Pharmacokinetics of novel plant cell-expressed taliglucerase alfa in adult and pediatric patients with Gaucher disease

Abbas¹,

Alon²,

Park³

et al. 2014

Molecular Genetics and Metabolism

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A pharmacokinetic analysis of a novel enzyme replacement therapy with Gene-Activated® human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease

Cited by 56 publications

References 8 publications

Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study

Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study

Pivotal trial with plant cell–expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease

Pharmacokinetics of novel plant cell-expressed taliglucerase alfa in adult and pediatric patients with Gaucher disease

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