ReviewDrugs2014
Eliglustat: first global approval.
Review in Drugs, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 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
21 citing papers in PubMed, 51 citations in OpenAlex.
- A comprehensive discovery platform for ELOVL1 small-molecule inhibitors targeting very long-chain fatty acid synthesis in adrenoleukodystrophy.The Journal of biological chemistry · 2026Article
- Lysosomal Neuraminidase 1 (NEU1): Its Unique Molecular Characters and Therapeutic Approaches for Deficiencies.Advances in experimental medicine and biology · 2026Review
- Elovl1 inhibition reduced very long chain fatty acids in a mouse model of adrenoleukodystrophy.iScience · 2025Article
- UDP-Glucose Ceramide Glucosyltransferase Inhibition, Immune Cell Mediation, and Endometriosis Risk: A Mendelian Randomization Study.International journal of women's health · 2025Article
- Allele frequency of pathogenic variants causing acid sphingomyelinase deficiency and Gaucher disease in the general Japanese population.Human genome variation · 2024Article
- Lysosomal sialidase NEU1, its intracellular properties, deficiency, and use as a therapeutic agent.Glycoconjugate journal · 2023Review
- Lipid metabolic vulnerabilities of multiple myeloma.Clinical and experimental medicine · 2023Review
- Modulating glycosphingolipid metabolism and autophagy improves outcomes in pre-clinical models of myeloma bone disease.Nature communications · 2022Article
- Total Synthesis of Eliglustat via Diastereoselective Amination of ChiralMolecules (Basel, Switzerland) · 2022Article
- RareMolecular genetics and metabolism reports · 2019Article
- Drug-Drug Interactions Of Amiodarone And Quinidine On The Pharmacokinetics Of Eliglustat In Rats.Drug design, development and therapy · 2019Article
- Lysosomal storage diseases.Nature reviews. Disease primers · 2018Review
- Attitudes of Individuals with Gaucher Disease toward Substrate Reduction Therapies.Journal of genetic counseling · 2018Article
- Induced pluripotent stem cell models of lysosomal storage disorders.Disease models & mechanisms · 2017Review
- Protection against Shiga Toxins.Toxins · 2017Review
- A peptide-linked recombinant glucocerebrosidase for targeted neuronal delivery: Design, production, and assessment.Journal of biotechnology · 2016Article
- Targeting glucosylceramide synthase upregulation reverts sorafenib resistance in experimental hepatocellular carcinoma.Oncotarget · 2016Article
- Article
- Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease.Drug design, development and therapy · 2015Review
- Article
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
Eliglustat [Cerdelga™ (US, EU)], a small-molecule oral glucosylceramide analogue that inhibits the enzyme glucosylceramide synthase has been developed by Genzyme Corporation (a subsidiary of Sanofi) for the treatment of Gaucher disease type 1 in adults. Inhibition of this enzyme reduces the accumulation of the lipid glucosylceramide in the liver, spleen, bone marrow and other organs. Eliglustat received its first global approval in this indication in the US, for use in treatment-naïve and treatment-experienced adult patients. It is also under regulatory review in the EU and Japan. This article summarizes the milestones in the development of eliglustat leading to this first approval for Gaucher disease type 1.
Indexed as
Identifiers
What OpenQuestion holds
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.