Page last updated: 2024-11-13

eliglustat tartrate

Description Research Excerpts Clinical Trials Roles Classes Pathways Study Profile Bioassays Related Drugs Related Conditions Protein Interactions Research Growth Market Indicators

Description

agalsidase beta: recombinant protein for treatment of Fabry disease [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

eliglustat tartrate : A tartrate that is the hemitartrate salt of eliglustat. A ceramide glucosyltransferase inhibitor used (as its tartrate salt) for treatment of Gaucher's disease. [Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Cross-References

ID SourceID
PubMed CID52918379
CHEBI ID83353
MeSH IDM0592239

Synonyms (49)

Synonym
104138-64-9
agalsidase beta
eliglustat tartrate
eliglustat hemitartrate
928659-70-5
eliglustat tartrate (jan/usan)
cerdelga (tn)
D09894
bis(n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(pyrrolidin-1- ylmethyl)ethyl)octanamide) (2r,3r)-2,3-dihydroxybutanedioate
genz 112638
eliglustat tartrate [usan]
octanamide, n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1- pyrrolidinylmethyl)ethyl)-, (2r,3r)-2,3-dihydroxybutanedioate (2:1)
n0493335p3 ,
cerdelga
unii-n0493335p3
(1r,2r)-octanoic acid(2-(2',3'-dihydro-benzo(1,4) dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl)-amide-l-tartaric acid salt
eliglustat tartrate [mi]
eliglustat tartrate [jan]
octanamide, n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl)-, (2r,3r)-2,3-dihydroxybutanedioate (2:1)
eliglustat tartrate [who-dd]
eliglustat tartrate [orange book]
eliglustat tartrate component of cerdelga
cerdelga component eliglustat tartrate
bis{n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl]octanamide} (2r,3r)-2,3-dihydroxysuccinic acid
CHEBI:83353 ,
bis{1-[(2r,3r)-3-(2,3-dihydro-1,4-benzodioxin-6-yl)-3-hydroxy-2-(octanoylamino)propyl]pyrrolidinium} (2r,3r)-2,3-dihydroxysuccinate
eliglustat l-tartrate
AC-35333
HY-14885A
eliglustat (hemitartrate)
CS-5423
DTXSID50239166 ,
genz-112638;eliglustat hemitartrate
eliglustat hemitartrate (genz-112638)
Q27156779
n-((1r,2r)-1-(2,3-dihydrobenzo[b][1,4]dioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide ((2r,3r)-2,3-dihydroxysuccinate)(2:1)
928659-70-5 (tartrate)
EX-A2301-1
genz-112638;eliglustat tartrate
n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-pyrrolidin-1-ylpropan-2-yl]octanamide;(2r,3r)-2,3-dihydroxybutanedioic acid
C16736
n-((1r,2r)-1-(2,3-dihydrobenzo[b][1,4]dioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide hemi((2r,3r)-2,3-dihydroxysuccinate)
MS-31794
eliglustat tartrategenz-112638
S4433
dtxcid70161657
n-((1r,2r)-1-(2,3-dihydrobenzo(b)(1,4)dioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide hemi-(2r,3r)-tartrate
bis(n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(pyrrolidin-1-ylmethyl)ethyl)octanamide) (2r,3r)-2,3-dihydroxybutanedioate
bis(1-((2r,3r)-3-(2,3-dihydro-1,4-benzodioxin-6-yl)-3-hydroxy-2-(octanoylamino)propyl)pyrrolidinium) (2r,3r)-2,3-dihydroxysuccinate

Research Excerpts

Toxicity

ExcerptReferenceRelevance
" Agalsidase beta was generally well tolerated; most treatment-related adverse events were mild or moderate infusion-associated reactions involving rigors, fever, or rhinitis."( Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease.
Germain, DP; Guffon, N; Lien, YH; Tsimaratos, M; Tylki-Szymanska, A; Vellodi, A; Wraith, JE, 2008
)
0.35
"To report a severe adverse event related to enzyme replacement therapy with agalsidase in an hemizygous male patient treated for Fabry disease."( Enzyme therapy in Fabry disease: severe adverse events associated with anti-agalsidase cross-reactive IgG antibodies.
Ackaert, C; Arnaud, P; Lidove, O; Nicaise, P; Papo, T; Reberga, A; Tesmoingt, C; Thetis, M, 2009
)
0.35
"Retrospective analysis of clinical, radiological and biochemical data in a patient who suffered adverse events related to both agalsidase alfa and agalsidase beta treatments."( Enzyme therapy in Fabry disease: severe adverse events associated with anti-agalsidase cross-reactive IgG antibodies.
Ackaert, C; Arnaud, P; Lidove, O; Nicaise, P; Papo, T; Reberga, A; Tesmoingt, C; Thetis, M, 2009
)
0.35
" Safety and efficacy evaluations included adverse drug reactions (ADRs), infusion-associated reactions and hypersensitivity reactions, and change in blood GL-3 level over time."( Long-term safety and efficacy of agalsidase beta in Japanese patients with Fabry disease: aggregate data from two post-authorization safety studies.
Hokugo, J; Suzuki, S; Tsurumi, M; Ueda, K, 2021
)
0.62
" Safety evaluation was performed including vital signs, laboratory tests, electrocardiograms, antibody screening tests, and adverse events at each visit."( A phase II, multicenter, open-label trial to evaluate the safety and efficacy of ISU303 (Agalsidase beta) in patients with Fabry disease.
Cheon, CK; Choi, JH; Choi, Y; Hwang, S; Kim, DS; Kim, JH; Kim, WS; Lee, BH; Lee, CH; Yoo, HW, 2022
)
0.72
" No severe adverse events were observed."( A phase II, multicenter, open-label trial to evaluate the safety and efficacy of ISU303 (Agalsidase beta) in patients with Fabry disease.
Cheon, CK; Choi, JH; Choi, Y; Hwang, S; Kim, DS; Kim, JH; Kim, WS; Lee, BH; Lee, CH; Yoo, HW, 2022
)
0.72
" These results suggest that ISU303 is safe and effective and can alternative ERT for FD."( A phase II, multicenter, open-label trial to evaluate the safety and efficacy of ISU303 (Agalsidase beta) in patients with Fabry disease.
Cheon, CK; Choi, JH; Choi, Y; Hwang, S; Kim, DS; Kim, JH; Kim, WS; Lee, BH; Lee, CH; Yoo, HW, 2022
)
0.72

Pharmacokinetics

ExcerptReferenceRelevance
" Here, migalastat and agalsidase beta biodistribution were assessed in mice and modeled using physiologically based pharmacokinetic (PBPK) analysis, and migalastat biodistribution was subsequently extrapolated to humans."( Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice.
Das, AM; Dungan, L; Garcia, A; Hamler, R; Johnson, FK; Khanna, R; Lun, Y; Martin, L; Perry, A; Schiffmann, R; Schmith, V; Shen, JS; Tsai, PC; Wu, YS, 2021
)
0.62

Compound-Compound Interactions

ExcerptReferenceRelevance
" Based on preclinical data, migalastat HCl in combination with agalsidase is expected to result in the pharmacokinetic (PK) enhancement of agalsidase in plasma by increasing the systemic exposure of active agalsidase, thereby leading to increased cellular levels in disease-relevant tissues."( Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase.
Adera, M; Barlow, C; Barth, J; Bichet, DG; Boudes, P; Eyskens, F; Flanagan, JJ; Goker-Alpan, O; Holida, M; Johnson, FK; Khanna, R; Lockhart, DJ; Nicholls, K; Shankar, S; Sitaraman, S; Thomas, M; Valenzano, KJ; Warnock, DG; Wustman, BA, 2015
)
0.42

Dosage Studied

ExcerptRelevanceReference
"The rechallenge infusion protocol specified strict patient monitoring conditions and graded dosing and infusion-rate schemes that were adjusted according to each patient's tolerance to the infusion."( Successful reinstitution of agalsidase beta therapy in Fabry disease patients with previous IgE-antibody or skin-test reactivity to the recombinant enzyme.
Bodensteiner, D; Cintron, RD; Germain, DP; Scott, CR; Shepherd, GM; Sims, KB, 2008
)
0.35
" The best model to explain seroconversion was a zero-inflated Poisson (ZIP) model where cumulative dose (under a constant dose regimen of dosing every 2 weeks) influenced the probability of seroconversion."( Conditional modeling of antibody titers using a zero-inflated poisson random effects model: application to Fabrazyme.
Bonate, PL; Richards, S; Sung, C; Welch, K, 2009
)
0.35
" The patient entered a phase III study of Replagal (agalsidase alfa) in 2001, allowing him to continue ERT with biweekly dosing for almost 10 years."( Progressive renal failure despite long-term biweekly enzyme replacement therapy in a patient with Fabry disease secondary to a new α-galactosidase mutation of Leu311Arg (L311R).
Imai, H; Kitagawa, W; Komatsuda, A; Miura, N; Nishikawa, K; Suzuki, K; Suzuki, S; Watanabe, D, 2011
)
0.37
" In summary, long-term enzyme replacement therapy in young patients can result in complete globotriaocylceramide clearance of mesangial and glomerular endothelial cells across all dosage regimens, and clearance of podocyte inclusions is dose-dependent."( Agalsidase benefits renal histology in young patients with Fabry disease.
Bostad, L; Hirth, A; Houge, G; Larsen, KK; Svarstad, E; Tøndel, C; Vikse, BE, 2013
)
0.39
" Furthermore, AB+ patients with dosage increase may experience new or increased IARs, necessitating in-hospital observations."( A revised home treatment algorithm for Fabry disease: influence of antibody formation.
Hollak, CE; Hoogendijk, SL; Linthorst, GE; Smid, BE; Wijburg, FA, 2013
)
0.39
"Two trials compared different dosing schedules of agalsidase alfa."( Enzyme replacement therapy for Anderson-Fabry disease.
Barreto, FC; Barretti, P; Bazan, R; Camargo, SE; Carvalho, RP; El Dib, R; Gomaa, H, 2016
)
0.43
"Our data suggest that a re-switch to high dosage of agalsidase results in a better biochemical response, but not in a significant renal amelioration especially in classical males."( Treatment switch in Fabry disease- a matter of dose?
Brand, E; Brand, SM; Canaan-Kühl, S; Duning, T; Kreul, L; Lenders, M; Lorenz, L; Nordbeck, P; Pogoda, C; Wanner, C, 2021
)
0.62
" Reduction of accumulated globotriaosylceramide (Gb3) and lysoGb3 in cultured Fabry fibroblasts by AGABIO and agalsidase beta showed comparable dose-response curves."( Development of a Biosimilar of Agalsidase Beta for the Treatment of Fabry Disease: Preclinical Evaluation.
Criscuolo, M; der Vlugt, KMMG; Gentilini, LD; Goorden, SMI; Hollak, CEM; Jacobs, M; Leen, R; Papouchado, M; Stet, FSB; Travella, A; van der Veen, S; van Kuilenburg, ABP, 2023
)
0.91
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Roles (1)

RoleDescription
EC 2.4.1.80 (ceramide glucosyltransferase) inhibitorAn EC 2.4.1.* (hexosyltransferase) inhibitor that interferes with the activity of ceramide glucosyltransferase (EC 2.4.1.80).
[role information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Drug Classes (1)

ClassDescription
tartrate saltA salt of the organic compound tartaric acid.
[compound class information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Research

Studies (220)

TimeframeStudies, This Drug (%)All Drugs %
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's97 (44.09)29.6817
2010's102 (46.36)24.3611
2020's21 (9.55)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 26.11

According to the monthly volume, diversity, and competition of internet searches for this compound, as well the volume and growth of publications, there is estimated to be moderate demand-to-supply ratio for research on this compound.

MetricThis Compound (vs All)
Research Demand Index26.11 (24.57)
Research Supply Index5.59 (2.92)
Research Growth Index4.51 (4.65)
Search Engine Demand Index34.37 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (26.11)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials24 (9.84%)5.53%
Reviews58 (23.77%)6.00%
Case Studies45 (18.44%)4.05%
Observational10 (4.10%)0.25%
Other107 (43.85%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]