ReviewDrugs2015
Eliglustat: A Review in Gaucher Disease Type 1.
Review in Drugs, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed, 43 citations in OpenAlex.
- Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects.Orphanet journal of rare diseases · 2017Trial
- The Sphingolipid Balance and Endothelial Dysfunction in Lysosomal Storage Diseases: Shared Mechanisms in Gaucher, Niemann-Pick and Fabry Disease.International journal of molecular sciences · 2026Review
- Article
- Experimental and MEDT Study of Sydnone-Alkyne Cycloaddition-Based Synthesis of 1,4-Disubstituted Pyrazoles and In Silico Investigation of Their Binding to HCV and HIV Proteins.Molecules (Basel, Switzerland) · 2026Article
- Overview of theLife (Basel, Switzerland) · 2025Review
- Current opinion on pluripotent stem cell technology in Gaucher's disease: challenges and future prospects.Cytotechnology · 2025Review
- Suicidal attempt with eliglustat overdose.JIMD reports · 2023Article
- Glucosylsphingosine (Lyso-GbInternational journal of molecular sciences · 2022Article
- Review
- Inhibition of P-Glycoprotein Does Not Increase the Efficacy of Proteasome Inhibitors in Multiple Myeloma Cells.ACS pharmacology & translational science · 2021Article
- Brain Penetrable Inhibitors of Ceramide Galactosyltransferase for the Treatment of Lysosomal Storage Disorders.ACS medicinal chemistry letters · 2020Article
- Impact of the organic cation transporter 2 inhibitor cimetidine on the single-dose pharmacokinetics of the glucosylceramide synthase inhibitor lucerastat in healthy subjects.European journal of clinical pharmacology · 2020Article
- Drug-Drug Interactions Of Amiodarone And Quinidine On The Pharmacokinetics Of Eliglustat In Rats.Drug design, development and therapy · 2019Article
- Small molecules as therapeutic agents for inborn errors of metabolism.Journal of inherited metabolic disease · 2017Review
- Pharmaceutical Chaperones and Proteostasis Regulators in the Therapy of Lysosomal Storage Disorders: Current Perspective and Future Promises.Frontiers in pharmacology · 2017Review
- Progress and potential of non-inhibitory small molecule chaperones for the treatment of Gaucher disease and its implications for Parkinson disease.Expert review of proteomics · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Oral eliglustat (Cerdelga®) is approved in several countries for the long-term treatment of adults with Gaucher disease type 1 (GD1) who are cytochrome P450 (CYP) 2D6 extensive metabolizers (EMs), intermediate metabolizer (IMs) or poor metabolizers (PMs) [these three CYP categories encompass >90 % of individuals]. Eliglustat is a potent, selective inhibitor of glucosylceramide synthase, the rate-limiting enzyme in the synthesis of certain glycosphingolipids, and thus, reduces the rate of biosynthesis of glycosphingolipids to counteract the catabolic defect (i.e. substrate reduction therapy). In the 9-month phase 3 ENGAGE trial, eliglustat significantly improved haematological endpoints and reduced organomegaly compared with placebo in treatment-naive adults with GD1, with the bone marrow burden score (a marker of Gaucher cell infiltration) and GD1 biomarkers also improving from baseline in eliglustat recipients. After 12 months in the phase 3 ENCORE trial, oral eliglustat was noninferior to intravenous imiglucerase [an enzyme replacement therapy (ERT)] in maintaining disease stability in adults who had stable disease after receiving ERT for ≥3 years. During long-term treatment with eliglustat (≤4 years) in the extension period of each of these pivotal trials and a phase 2 trial, patients experienced sustained improvements in visceral, haematological and skeletal endpoints, with no new safety concerns identified. Further clinical experience will help to more definitively establish the position of eliglustat treatment in adults with GD1. In the meantime, with its convenient oral regimen, eliglustat is an emerging alternative therapy to ERT for the long-term treatment of adults with GD1 who are CYP2D6 EMs, IMs or PMs.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.