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Volumn 2, Issue 5, 2014, Pages 523-529

A systematic review on effectiveness and safety of eliglustat for type 1 Gaucher disease

Author keywords

Eliglustat (tartrate); Gaucher disease; Lysosomal storage disorder; Substrate reduction therapy

Indexed keywords

CYTOCHROME P450 2D6; ELIGLUSTAT; HEMOGLOBIN; IMIGLUCERASE; MIGLUSTAT; PLACEBO;

EID: 84899070142     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2014.899148     Document Type: Article
Times cited : (12)

References (45)
  • 1
    • 50549198437 scopus 로고
    • Metabolism of glucocerebrosides II Evidence of an enzymatic deficiency in Gaucher disease
    • Brady RO, Kanfer JN, Shapiro D. Metabolism of glucocerebrosides.II. Evidence of an enzymatic deficiency in Gaucher disease. Biochem Biophys Res Commun 1965;18:221-5
    • (1965) Biochem Biophys Res Commun , vol.18 , pp. 221-225
    • Brady, R.O.1    Kanfer, J.N.2    Shapiro, D.3
  • 2
    • 0025869216 scopus 로고
    • Replacement therapy for inherited enzyme deficiency: Macrophage-Targeted glucocerebrosidase for gaucher's disease
    • Barton NW, Brady RO, Dambrosia JM, et al. Replacement therapy for inherited enzyme deficiency: Macrophage-Targeted glucocerebrosidase for gaucher's disease. N Engl J Med 1991;324:1464-70
    • (1991) N Engl J Med , vol.324 , pp. 1464-1470
    • Barton, N.W.1    Brady, R.O.2    Dambrosia, J.M.3
  • 3
    • 0025236339 scopus 로고
    • Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with gaucher disease
    • Barton NW, Furbish FS, Murray GJ, et al. Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with gaucher disease. Proc Natl Acad Sci USA 1990;87:1913-16
    • (1990) Proc Natl Acad Sci USA , vol.87 , pp. 1913-1916
    • Barton, N.W.1    Furbish, F.S.2    Murray, G.J.3
  • 4
    • 0026014938 scopus 로고
    • Enzyme replacement therapy for gaucher disease
    • Beutler E, Kay A, Saven A, et al. Enzyme replacement therapy for gaucher disease. Blood 1991;78:1183-9
    • (1991) Blood , vol.78 , pp. 1183-1189
    • Beutler, E.1    Kay, A.2    Saven, A.3
  • 5
    • 0028883136 scopus 로고
    • Enzyme therapy in type 1 gaucher disease: Comparative efficacy of mannoseterminated glucocerebrosidase from natural and recombinant sources
    • Grabowski GA, Barton NW, Pastores G, et al. Enzyme therapy in type 1 gaucher disease: Comparative efficacy of mannoseterminated glucocerebrosidase from natural and recombinant sources. Ann Intern Med 1995;122:33-9
    • (1995) Ann Intern Med , vol.122 , pp. 33-39
    • Grabowski, G.A.1    Barton, N.W.2    Pastores, G.3
  • 6
    • 84874302849 scopus 로고    scopus 로고
    • Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, phase 3 study
    • Gonzalez DE, Turkia HB, Lukina EA, et al. Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, phase 3 study. Am J Hematol 2013;88:166-71
    • (2013) Am J Hematol , vol.88 , pp. 166-171
    • Gonzalez, D.E.1    Turkia, H.B.2    Lukina, E.A.3
  • 7
    • 77954693904 scopus 로고    scopus 로고
    • Phase I/II and extension study of velaglucerase alfa replacement therapy in adults with type 1 gaucher disease: 48-month experience
    • Zimran A, Altarescu G, Phillips M, et al. Phase I/II and extension study of velaglucerase alfa replacement therapy in adults with type 1 gaucher disease: 48-month experience. Blood 2010;115:4651-46
    • (2010) Blood , vol.115 , pp. 4651-4646
    • Zimran, A.1    Altarescu, G.2    Phillips, M.3
  • 8
    • 82155184565 scopus 로고    scopus 로고
    • Pivotal trial with plant cellexpressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for gaucher disease
    • Zimran A, Brill-Almon E, Chertkoff R, et al. Pivotal trial with plant cellexpressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for gaucher disease. Blood 2011;118:5767-73
    • (2011) Blood , vol.118 , pp. 5767-5773
    • Zimran, A.1    Brill-Almon, E.2    Chertkoff, R.3
  • 9
    • 46849112025 scopus 로고    scopus 로고
    • Persistent bone disease in adult type 1 gaucher disease despite increasing doses of enzyme replacement therapy
    • de Fost M, van Noesel CJ, Aerts JM, et al. Persistent bone disease in adult type 1 gaucher disease despite increasing doses of enzyme replacement therapy. Haematologica 2008;93:1119-20
    • (2008) Haematologica , vol.93 , pp. 1119-1120
    • De Fost, M.1    Van Noesel, C.J.2    Aerts, J.M.3
  • 10
    • 0029939988 scopus 로고    scopus 로고
    • Treatment of Gaucher disease with an enzyme inhibitor
    • Radin NS. Treatment of Gaucher disease with an enzyme inhibitor. Glycoconj J 1996;13:153-7
    • (1996) Glycoconj J , vol.13 , pp. 153-157
    • Radin, N.S.1
  • 11
    • 77958483334 scopus 로고    scopus 로고
    • Pharmacological small molecules for the treatment of lysosomal storage disorders
    • Smid BE, Aerts JMFG, Boot RG, et al. Pharmacological small molecules for the treatment of lysosomal storage disorders. Expert Opin Investig Drugs 2010;19:1367-79
    • (2010) Expert Opin Investig Drugs , vol.19 , pp. 1367-1379
    • Smid, B.E.1    Aerts, J.M.F.G.2    Boot, R.G.3
  • 12
    • 10744226382 scopus 로고    scopus 로고
    • The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) gaucher disease: A position statement
    • Cox TM, Aerts JM, Andria G, et al. The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) gaucher disease: A position statement. J Inherit Metab Dis 2003;26:513-26
    • (2003) J Inherit Metab Dis , vol.26 , pp. 513-526
    • Cox, T.M.1    Aerts, J.M.2    Andria, G.3
  • 13
    • 0034728914 scopus 로고    scopus 로고
    • Novel oral treatment of gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis
    • Cox T, Lachmann R, Hollak C, et al. Novel oral treatment of gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Lancet 2000;355:1481-5
    • (2000) Lancet , vol.355 , pp. 1481-1485
    • Cox, T.1    Lachmann, R.2    Hollak, C.3
  • 14
    • 84871603938 scopus 로고    scopus 로고
    • Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 gaucher disease: A prospective, open-label non-inferiority study
    • Cox TM, Amato D, Hollak CE, et al. Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 gaucher disease: A prospective, open-label non-inferiority study. Orphanet J Rare Dis 2012;7:102
    • (2012) Orphanet J Rare Dis , vol.7 , pp. 102
    • Cox, T.M.1    Amato, D.2    Hollak, C.E.3
  • 15
    • 34249306894 scopus 로고    scopus 로고
    • A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of gaucher disease
    • McEachern KA, Fung J, Komarnitsky S, et al. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of gaucher disease. Mol Genet Metab 2007;91:259-67
    • (2007) Mol Genet Metab , vol.91 , pp. 259-267
    • McEachern, K.A.1    Fung, J.2    Komarnitsky, S.3
  • 16
    • 0030814767 scopus 로고    scopus 로고
    • Extensive glycosphingolipid depletion in the liver and lymphoid organs of mice treated with N-butyldeoxynojirimycin
    • Platt FM, Reinkensmeier G, Dwek RA, Butters TD. Extensive glycosphingolipid depletion in the liver and lymphoid organs of mice treated with N-butyldeoxynojirimycin. J Biol Chem 1997;272:19365-72
    • (1997) J Biol Chem , vol.272 , pp. 19365-19372
    • Platt, F.M.1    Reinkensmeier, G.2    Dwek, R.A.3    Butters, T.D.4
  • 17
    • 0033591332 scopus 로고    scopus 로고
    • Improved inhibitors of glucosylceramide synthase
    • Lee L, Abe A, Shayman JA. Improved inhibitors of glucosylceramide synthase. J Biol Chem 1999;274:14662-9
    • (1999) J Biol Chem , vol.274 , pp. 14662-14669
    • Lee, L.1    Abe, A.2    Shayman, J.A.3
  • 18
    • 84952989751 scopus 로고    scopus 로고
    • Eliglustat tartrate: Glucosylceramide synthase inhibitor treatment of type 1 gaucher disease
    • Shayman JA. Eliglustat tartrate: Glucosylceramide synthase inhibitor treatment of type 1 gaucher disease. Drugs Future 2010;35:613-20
    • (2010) Drugs Future , vol.35 , pp. 613-620
    • Shayman, J.A.1
  • 19
    • 84895855941 scopus 로고    scopus 로고
    • The design and clinical development of inhibitors of glycosphingolipid syn thesis: Will invention be the mother of necessity?
    • Shayman JA. The design and clinical development of inhibitors of glycosphingolipid synthesis: Will invention be the mother of necessity? Trans Am Clin Climatol Assoc 2013;124:46-60
    • (2013) Trans Am Clin Climatol Assoc , vol.124 , pp. 46-60
    • Shayman, J.A.1
  • 20
    • 0034091578 scopus 로고    scopus 로고
    • Reduction of globotriaosylceramide in fabry disease mice by substrate deprivation
    • Abe A, Gregory S, Lee L, et al. Reduction of globotriaosylceramide in fabry disease mice by substrate deprivation. J Clin Invest 2000;105:1563-71
    • (2000) J Clin Invest , vol.105 , pp. 1563-1571
    • Abe, A.1    Gregory, S.2    Lee, L.3
  • 21
    • 84863399238 scopus 로고    scopus 로고
    • Property-based design of a glucosylceramide synthase inhibitor that reduces glucosylceramide in the brain
    • Larsen SD, Wilson MW, Abe A, et al. Property-based design of a glucosylceramide synthase inhibitor that reduces glucosylceramide in the brain. J Lipid Res 2012;53:282-91
    • (2012) J Lipid Res , vol.53 , pp. 282-291
    • Larsen, S.D.1    Wilson, M.W.2    Abe, A.3
  • 22
    • 77957601016 scopus 로고    scopus 로고
    • Safety, tolerability, and pharmacokinetics of eliglustat tartrate (Genz-112638) after single doses, multiple doses, and food in healthy volunteers
    • Peterschmitt MJ, Burke A, Blankstein L, et al. Safety, tolerability, and pharmacokinetics of eliglustat tartrate (Genz-112638) after single doses, multiple doses, and food in healthy volunteers. J Clin Pharmacol 2011;51:695-705
    • (2011) J Clin Pharmacol , vol.51 , pp. 695-705
    • Peterschmitt, M.J.1    Burke, A.2    Blankstein, L.3
  • 23
    • 77954538917 scopus 로고    scopus 로고
    • A Phase 2 study of eliglustat tartrate (Genz-112638) an oral substrate reduction therapy for Gaucher disease type 1
    • Lukina E, Watman N, Arreguin EA, et al. A Phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Blood 2010;116(6):893-9
    • (2010) Blood , vol.116 , Issue.6 , pp. 893-899
    • Lukina, E.1    Watman, N.2    Arreguin, E.A.3
  • 24
    • 77957551053 scopus 로고    scopus 로고
    • Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study
    • Lukina E, Watman N, Arreguin EA, et al. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood 2010;116:4095-8
    • (2010) Blood , vol.116 , pp. 4095-4098
    • Lukina, E.1    Watman, N.2    Arreguin, E.A.3
  • 25
    • 84899120995 scopus 로고    scopus 로고
    • Eliglustat, an investigational oral therapy for gaucher disease type 1: Phase 2 results after 4 years of treatment
    • Conference: 54th Annual Meeting of the American Society of Hematology, ASH
    • Peterschmitt MJ, Lukina E, Watman N, et al. Eliglustat, an investigational oral therapy for gaucher disease type 1: Phase 2 results after 4 years of treatment. Blood. Conference: 54th Annual Meeting of the American Society of Hematology, ASH; 2012
    • (2012) Blood
    • Peterschmitt, M.J.1    Lukina, E.2    Watman, N.3
  • 27
    • 84899067946 scopus 로고    scopus 로고
    • Improvements in skeletal manifestations in Gaucher disease type 1 patients after 3 years of treatment with oral eliglustat during a phase 2 trial
    • Peterschmitt MJ, Kamath RS, Lukina E, et al. Improvements in skeletal manifestations in Gaucher disease type 1 patients after 3 years of treatment with oral eliglustat during a phase 2 trial. 53rd Annual Meeting of the American Society of Hematology, ASH; 2011
    • (2011) 53rd Annual Meeting of the American Society of Hematology ASH
    • Peterschmitt, M.J.1    Kamath, R.S.2    Lukina, E.3
  • 28
    • 84899096152 scopus 로고    scopus 로고
    • Biomarker responses after 3 years of treatment with eliglustat, an investigational oral therapy for gaucher disease type 1 (GD1)
    • 29. Available from [Last accessed 12 June 2010]
    • Puga AC, Lukina E, Watman N, et al. Biomarker responses after 3 years of treatment with eliglustat, an investigational oral therapy for gaucher disease type 1 (GD1). Annual symposium of the Society for the Study of Inborn Errors of Metabolism; 2011 29. Available from: Www.clinicaltrials.gov [Last accessed 12 June 2010]
    • (2011) Annual symposium of the Society for the Study of Inborn Errors of Metabolism
    • Puga, A.C.1    Lukina, E.2    Watman, N.3
  • 29
    • 84899045692 scopus 로고    scopus 로고
    • Available from
    • Available from: Http://www.ctri.nic.in/ Clinicaltrials/pmaindet2.php? trialid=875
  • 30
    • 84899099101 scopus 로고    scopus 로고
    • ENGAGE: A phase 3 randomized double-blind placebo-controlled multi-center study to investigate the efficacy and safety of eliglustat in adults with gaucher disease type 1 (GD1): 9 month results
    • Available from
    • Packman S, Amato D, Dasouki M, et al. ENGAGE: A phase 3, randomized, double-blind, placebo-controlled, multi-center study to investigate the efficacy and safety of eliglustat in adults with gaucher disease type 1 (GD1): 9 month results. ASHG; 2013. Available from: Http://abstracts.ashg.org/ cgi-bin/2013/ashg13s.pl? author=packman&sort= ptimes&sbutton= Detail&absno=130122683&sid=571755
    • (2013) ASHG
    • Packman, S.1    Amato, D.2    Dasouki, M.3
  • 31
    • 84899072640 scopus 로고    scopus 로고
    • Available from Last accessed 18 November 2013]
    • Available from: Http://news.genzyme. com/press-release/genzyme- Announcespositive-new-data-Two-phase-3-studiesoral-eliglustat-Tartrate-gauch [Last accessed 18 November 2013]
  • 32
    • 78049506152 scopus 로고    scopus 로고
    • Short-Term withdrawal from imiglucerase: What can we learn from it?
    • Hollak CEM. Short-Term withdrawal from imiglucerase: What can we learn from it? Blood Cells Mol Dis 2011;46:105-6
    • (2011) Blood Cells Mol Dis , vol.46 , pp. 105-106
    • Hollak, C.E.M.1
  • 33
    • 78650819782 scopus 로고    scopus 로고
    • Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply
    • Zimran A, Altarescu G, Elstein D. Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply. Blood Cells Mol Dis 2011;46:111-14
    • (2011) Blood Cells Mol Dis , vol.46 , pp. 111-114
    • Zimran, A.1    Altarescu, G.2    Elstein, D.3
  • 34
    • 78650816915 scopus 로고    scopus 로고
    • Enzyme replacement therapy 'drug holiday' results from an unexpected shortage of an orphan drug supply in Australia
    • Goldblatt J, Fletcher JM, McGill J, et al. Enzyme replacement therapy 'drug holiday': Results from an unexpected shortage of an orphan drug supply in australia. Blood Cells Mol Dis 2011;46:107-10
    • (2011) Blood Cells Mol Dis , vol.46 , pp. 107-110
    • Goldblatt, J.1    Fletcher, J.M.2    McGill, J.3
  • 35
    • 84899121680 scopus 로고    scopus 로고
    • ENCORE: A randomized, controlled, open-label non-inferiority study comparing eliglustat to imiglucerase in gaucher disease type 1 patients on enzyme replacement therapy who have reached therapeutic goals
    • Available from
    • Balwani M, Cox TM, Drelichman G, et al. ENCORE: A randomized, controlled, open-label non-inferiority study comparing eliglustat to imiglucerase in gaucher disease type 1 patients on enzyme replacement therapy who have reached therapeutic goals. American Society of Human Genetics; 2013. Available from: Http://www.ashg.org/2013meeting/ abstracts/fulltext/f130122761.htm
    • (2013) American Society of Human Genetics
    • Balwani, M.1    Cox, T.M.2    Drelichman, G.3
  • 36
    • 84899099777 scopus 로고    scopus 로고
    • Encore: A randomized, controlled, open-label non-inferiority study comparing eliglustat to imiglucerase in gaucher disease type 1 patients on enzyme replacement therapy who have reached therapeutic goals abstract of worldsymposium 2014
    • Cox TM, Drelichman G, Cravo R, et al. ENCORE: A randomized, controlled, open-label non-inferiority study comparing eliglustat to imiglucerase in gaucher disease type 1 patients on enzyme replacement therapy who have reached therapeutic goals. Abstract of WORLDsymposium 2014. Mol Genet Metab 2014;111:S1-S118
    • (2014) Mol Genet Metab , vol.111
    • Cox, T.M.1    Drelichman, G.2    Cravo, R.3
  • 37
    • 84878496452 scopus 로고    scopus 로고
    • Malignancies and monoclonal gammopathy in Gaucher disease; a systematic review of the literature
    • Arends M, van DL, Biegstraaten M, Hollak CE. Malignancies and monoclonal gammopathy in Gaucher disease; a systematic review of the literature. Br J Haematol 2013;161:832-42
    • (2013) Br J Haematol , vol.161 , pp. 832-842
    • Arends, M.1    Van, D.L.2    Biegstraaten, M.3    Hollak, C.E.4
  • 38
    • 40849113048 scopus 로고    scopus 로고
    • Type I gaucher disease, a glycosphingolipid storage disorder, is associated with insulin resistance
    • Langeveld M, Ghauharali KJ, Sauerwein HP, et al. Type I gaucher disease, a glycosphingolipid storage disorder, is associated with insulin resistance. J Clin Endocrinol Metab 2008;93:845-51
    • (2008) J Clin Endocrinol Metab , vol.93 , pp. 845-851
    • Langeveld, M.1    Ghauharali, K.J.2    Sauerwein, H.P.3
  • 39
    • 0029773625 scopus 로고    scopus 로고
    • Occurrence of parkinson's syndrome in type I gaucher disease
    • Neudorfer O, Giladi N, Elstein D, et al. Occurrence of parkinson's syndrome in type I gaucher disease. QJM 1996;89:691-4
    • (1996) QJM , vol.89 , pp. 691-694
    • Neudorfer, O.1    Giladi, N.2    Elstein, D.3
  • 40
    • 84885139621 scopus 로고    scopus 로고
    • B cell lymphoma and myeloma in murine Gaucher's disease
    • Pavlova EV, Wang SZ, Archer J, et al. B cell lymphoma and myeloma in murine Gaucher's disease. J Pathol 2013;231:88-97
    • (2013) J Pathol , vol.231 , pp. 88-97
    • Pavlova, E.V.1    Wang, S.Z.2    Archer, J.3
  • 41
    • 79955119883 scopus 로고    scopus 로고
    • Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders
    • Hollak CE, Aerts JM, Ayme S, Manuel J. Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders. Orphanet J Rare Dis 2011;6:16
    • (2011) Orphanet J Rare Dis , vol.6 , pp. 16
    • Hollak, C.E.1    Aerts, J.M.2    Ayme, S.3    Manuel, J.4
  • 42
    • 84899118543 scopus 로고    scopus 로고
    • Modelling gaucher disease progression: Long term enzyme replacement therapy reduces the incidence of splenectomy and bone complications thesis univeristy of amsterdam two decades of treatment for gaucher disease
    • van Dussen L, Biegstraaten M, Dijkgraaf MGW, Hollak CE. Modelling gaucher disease progression: Long term enzyme replacement therapy reduces the incidence of splenectomy and bone complications. Thesis Univeristy of Amsterdam: Two decades of treatment for gaucher disease. Amsterdam; 2014
    • (2014) Amsterdam
    • Van Dussen, L.1    Biegstraaten, M.2    Dijkgraaf, M.G.W.3    Hollak, C.E.4
  • 43
    • 77957680068 scopus 로고    scopus 로고
    • Peripheral neuropathy in adult type 1 gaucher disease: A 2-year prospective observational study
    • Biegstraaten M, Mengel E, Marodi L, et al. Peripheral neuropathy in adult type 1 gaucher disease: A 2-year prospective observational study. Brain 2010;133:2909-19
    • (2010) Brain , vol.133 , pp. 2909-2919
    • Biegstraaten, M.1    Mengel, E.2    Marodi, L.3
  • 44
    • 70450173661 scopus 로고    scopus 로고
    • Miglustat (Zavesca) in type 1 gaucher disease: 5-year results of a post-Authorisation safety surveillance programme
    • Hollak CE, Hughes D, van Schaik IN, et al. Miglustat (Zavesca) in type 1 gaucher disease: 5-year results of a post-Authorisation safety surveillance programme. Pharmacoepidemiol Drug Saf 2009;18:770-7
    • (2009) Pharmacoepidemiol Drug Saf , vol.18 , pp. 770-777
    • Hollak, C.E.1    Hughes, D.2    Van Schaik, I.N.3
  • 45
    • 84899047612 scopus 로고    scopus 로고
    • Eliglustat safety profile based on a pooled analysis of data from four trials in gaucher disease type i abstract of worldsymposium 2014
    • Ross L, Peterschmitt J, Puga AC, et al. Eliglustat safety profile based on a pooled analysis of data from four trials in gaucher disease type I. Abstract of WORLDsymposium 2014. Mol Genet Metab 2014;111:S1-S118
    • (2014) Mol Genet Metab , vol.111
    • Ross, L.1    Peterschmitt, J.2    Puga, A.C.3


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