Eliglustat

Summary

Eliglustat (CHEMBL2110588) is an approved small-molecule EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor (ATC A16AX10); indicated across 4 conditions including gaucher disease and hematopoietic and lymphoid system neoplasm.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A16AX10
• Indications: 4 conditions
• Clinical trials: 14
• Chemistry: 404.5 Da · C23H36N2O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2110588
Name	Eliglustat
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	23652731
ChEBI	CHEBI:82752
ATC	A16AX10
Molecular formula	C23H36N2O4
Molecular weight	404.5
InChIKey	FJZZPCZKBUKGGU-AUSIDOKSSA-N

SMILES: CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O

IUPAC name: N-[(1R,2R)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-pyrrolidin-1-ylpropan-2-yl]octanamide

ChEBI definition: A carboxamide obtained by formal condensation of the carboxy group of octanoic acid with the primary amino group of (1R,2R)-2-amino-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-3-(pyrrolidin-1-yl)propan-1-ol. A ceramide glucosyltransferase inhibitor used (as its tartrate salt) for treatment of Gaucher’s disease.

Pharmacological roles (ChEBI): EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor.

Also known as: Eliglustat, GENZ 99067, GENZ-99067, ELIGLUSTAT, eliglustat

Parent form; salt/anhydrous children: CHEMBL4297066, CHEMBL5723563

Patent coverage: 285 distinct patent families (687 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 675 (98%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 4 (assay-derived). Sample: Ceramide glucosyltransferase, 5-hydroxytryptamine receptor 1A, Prostaglandin G/H synthase 1, Mu-type opioid receptor.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 5 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
UGCG	7.8	IC50	16	nM	CHEMBL_ACT_15663586
PTGS1	5	AC50	10050	nM	CHEMBL_ACT_25205535

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

4 indications (3 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
Gaucher disease	4	MONDO:0018150	Orphanet:77259
hematopoietic and lymphoid system neoplasm	1	MONDO:0002334	MONDO:0044881

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 14.

Phase distribution

Phase	Trials
PHASE1	8
PHASE3	2
PHASE2	1
PHASE1/PHASE2	1
EARLY_PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02536755	PHASE3	COMPLETED	Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
NCT03485677	PHASE3	COMPLETED	Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
NCT06558773	PHASE2	RECRUITING	GSL Synthetase Inhibitor Plus Immune Checkpoint Inhibitor and/or Regorafenib in Previously Treated pMMR/MSS CRC.
NCT07138547	PHASE1/PHASE2	RECRUITING	GSL Synthetase Inhibitor Eliglustat Combined With CD30 Target Immunotherapy for the Treatment of of CD30+ Lymphoma
NCT01452542	PHASE1	COMPLETED	A Phase I Study of the Pharmacokinetic Variability and Relative Bioavailability of the Phase 3 and Common Blend Formulations of Eliglustat in Healthy Adult Subjects
NCT01659944	PHASE1	COMPLETED	Study to Evaluate the Effect of Eliglustat on the Pharmacokinetics, Safety and Tolerability of Metoprolol in Healthy Adults
NCT02422654	PHASE1	COMPLETED	Taste Evaluation of Different Liquid Formulations With Eliglustat
NCT02536911	PHASE1	COMPLETED	A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
NCT02536937	PHASE1	COMPLETED	A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
NCT04944888	PHASE1	COMPLETED	GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor in Previously Treated Blood and Solid Tumor
NCT06188325	PHASE1	COMPLETED	A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers
NCT06193304	PHASE1	COMPLETED	A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth
NCT07090538	EARLY_PHASE1	NOT_YET_RECRUITING	GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma.
NCT03519646	Not specified	COMPLETED	Eliglustat on Gaucher Disease Type IIIB

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

PharmGKB dosing guidelines (1) — CPIC / DPWG genotype-guided dosing for this drug (drug × pharmacogene):

Guideline	Source	Gene(s)	Dosing	Recommendation
Annotation of DPWG Guideline for eliglustat and CYP2D6	DPWG	CYP2D6	yes	yes

PharmGKB also curates 0 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).