Miglustat

Summary

Miglustat (CHEMBL1029) is an approved small-molecule EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor (ATC A16AX06) targeting UGCG; indicated across 13 conditions including gaucher disease and niemann-pick disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A16AX06
• Targets: 1 (UGCG)
• Indications: 13 conditions
• Clinical trials: 24
• Chemistry: 219.28 Da · C10H21NO4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1029
Name	Miglustat
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	51634
ChEBI	CHEBI:50381
ATC	A16AX06
Molecular formula	C10H21NO4
Molecular weight	219.28
InChIKey	UQRORFVVSGFNRO-UTINFBMNSA-N

SMILES: CCCCN1C[C@@H]([C@H]([C@@H]([C@H]1CO)O)O)O

IUPAC name: (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol

ChEBI definition: A hydroxypiperidine that is deoxynojirimycin in which the amino hydrogen is replaced by a butyl group.

Pharmacological roles (ChEBI): EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor, anti-HIV agent.

Also known as: Butyldeoxynojirimycin, Miglustat, Miglustat dipharma, Miglustat gen.orph, N-butyldeoxynojirimycin, OGT 918, OGT-918, Opfolda, Yargesa, Zavesca, zavesca, SC-48334

Parent form; salt/anhydrous children: CHEMBL1329690

Patent coverage: 1,088 distinct patent families (4,770 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
UGCG	UDP-glucose ceramide glucosyltransferase	Inhibition	5.13	8.4%	Q16739

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Lysosomal alpha-glucosidase, Ceramide glucosyltransferase, Maltase-glucoamylase, Lysosomal alpha-glucosidase, Sucrase-isomaltase, intestinal, Sucrase-isomaltase, intestinal, Lysosomal alpha-glucosidase, Beta-glucosidase, Glycogen debranching enzyme, Non-lysosomal glucosylceramidase.

Bioactivity

ChEMBL activities: 23 potent at pChembl ≥ 5 of 35 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
GBA2	7.85	IC50	14	nM	CHEMBL_ACT_19223143
GAA	7	IC50	100	nM	CHEMBL_ACT_2947461
GAA	7	IC50	100	nM	CHEMBL_ACT_3204094
Q69ZF3	6.85	IC50	140	nM	CHEMBL_ACT_2186708
GAA	6.7	Ki	200	nM	CHEMBL_ACT_24394162
GBA2	6.64	IC50	230	nM	CHEMBL_ACT_10837264
GBA2	6.64	IC50	230	nM	CHEMBL_ACT_15096306
GBA2	6.64	IC50	230	nM	CHEMBL_ACT_2947464
GBA2	6.64	IC50	230	nM	CHEMBL_ACT_3204081
GBA2	6.64	IC50	230	nM	CHEMBL_ACT_5153775
GBA2	6.52	IC50	300	nM	CHEMBL_ACT_12103995
SI	6.3	IC50	500	nM	CHEMBL_ACT_3204030
P23739	6.24	IC50	580	nM	CHEMBL_ACT_451708
Q6P7A9	5.68	IC50	2100	nM	CHEMBL_ACT_12148463
P23739	5.68	IC50	2100	nM	CHEMBL_ACT_1461217
MGAM	5.68	IC50	2100	nM	CHEMBL_ACT_451707
P23739	5.57	IC50	2700	nM	CHEMBL_ACT_12148457
P23739	5.57	IC50	2700	nM	CHEMBL_ACT_1461316
P23739	5.57	IC50	2700	nM	CHEMBL_ACT_451709
Q6P7A9	5.28	IC50	5300	nM	CHEMBL_ACT_18106456
MGAM	5.05	IC50	9000	nM	CHEMBL_ACT_3204043
P70699	5.05	IC50	9000	nM	CHEMBL_ACT_5153697
AGL	5	IC50	10000	nM	CHEMBL_ACT_3204106

Target pathways

Aggregated over 1 target gene(s): UGCG.

Top Reactome pathways

5 total, by targets touching each:

Pathway	Targets	Genes
Metabolism	1	UGCG
Glycosphingolipid metabolism	1	UGCG
Sphingolipid metabolism	1	UGCG
Metabolism of lipids	1	UGCG
Glycosphingolipid biosynthesis	1	UGCG

Dominant GO biological processes

GO term	Targets
protein lipidation	1
glucosylceramide biosynthetic process	1
glycosphingolipid biosynthetic process	1
epidermis development	1
regulation of signal transduction	1
cell differentiation	1
keratinocyte differentiation	1
leptin-mediated signaling pathway	1
neuron development	1
establishment of skin barrier	1
intestinal lipid absorption	1
cornified envelope assembly	1
lipid metabolic process	1
sphingolipid metabolic process	1

Indications & clinical

Indications

13 indications (4 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
Niemann-Pick disease	4	MONDO:0001982	EFO:1001380
disorder of glycogen metabolism	3	MONDO:0002412	MONDO:0002412
Sandhoff disease	3	MONDO:0010006	MONDO:0010006
GM2 gangliosidosis	3	MONDO:0017720	MONDO:0017720
Niemann-Pick disease type C	3	MONDO:0018982	MONDO:0018982
HIV infectious disease	2	MONDO:0005109	EFO:0000764
cystic fibrosis	2	MONDO:0009061	MONDO:0009061
hereditary spastic paraplegia	2	MONDO:0019064	MONDO:0019064
diarrheal disease	1	MONDO:0001673	HP:0002014

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

Clinical trials

Total trials: 24.

Phase distribution

Phase	Trials
PHASE2	9
PHASE3	7
PHASE4	3
Not specified	3
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00194649	PHASE4	COMPLETED	Glycosphingolipid Inhibition and Spermatogenesis in Man: A Pilot Study (MIG 2)
NCT02030015	PHASE4	TERMINATED	Synergistic Enteral Regimen for Treatment of the Gangliosidoses
NCT03910621	PHASE4	COMPLETED	Safety and Efficacy of Miglustat in Chinese NPC Patients
NCT03911505	PHASE3	ACTIVE_NOT_RECRUITING	ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
NCT04808505	PHASE3	RECRUITING	A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
NCT00319046	PHASE3	COMPLETED	Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
NCT00672022	PHASE3	COMPLETED	Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
NCT01760564	PHASE3	COMPLETED	Application of Miglustat in Patients With Niemann-Pick Type C
NCT03729362	PHASE3	COMPLETED	A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
NCT03822013	PHASE3	TERMINATED	Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)
NCT00001993	PHASE2	COMPLETED	Initial Phase II Efficacy and Safety Study of SC-48334 Administered in Combination With Low-Dose Zidovudine (AZT) to Symptomatic HIV-1 Infected Patients With = or > 200 to = or < 500 CD4+ Cells/mm3
NCT00002079	PHASE2	COMPLETED	Phase II Study of the Safety and Surrogate Marker Efficacy of Butyldeoxynojirimycin (SC-48334) and AZT in Symptomatic HIV-1 Infected Patients With 200 - 500 CD4+ Cells/mm3. (NOTE: Asymptomatic HIV-1 Infected Patients Also Eligible)
NCT00041535	PHASE2	COMPLETED	OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
NCT00418847	PHASE2	COMPLETED	Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
NCT00517153	PHASE2	COMPLETED	Miglustat in Niemann-Pick Type C Disease
NCT00537602	PHASE2	TERMINATED	Miglustat / OGT 918 in the Treatment of Cystic Fibrosis
NCT00742092	PHASE2	COMPLETED	Miglustat in Cystic Fibrosis
NCT00945347	PHASE2	COMPLETED	Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?
NCT04768166	PHASE2	COMPLETED	Testing Miglustat Administration in Subjects With Spastic Paraplegia 11
NCT05174039	PHASE1/PHASE2	COMPLETED	An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease
NCT00000692	PHASE1	COMPLETED	Phase I Rising Dose Tolerability Study of SC-48334 in Patients With Acquired Immunodeficiency Syndrome (AIDS) and Advanced AIDS Related Complex
NCT06121011	Not specified	RECRUITING	A Global Prospective Observational Registry of Patients With Pompe Disease
NCT01451879	Not specified	COMPLETED	Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
NCT02520934	Not specified	UNKNOWN	Miglustat on Gaucher Disease Type IIIB

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

6 molecules share ≥1 primary target. Top 6 by shared-target count:

Molecule	Source	Status	Shared targets
ELIGLUSTAT	ChEMBL + PubChem	Phase 4 (approved)	UGCG
LUCERASTAT	ChEMBL	Phase 3	UGCG
VENGLUSTAT	ChEMBL	Phase 3	UGCG
NIZUBAGLUSTAT	ChEMBL	Phase 2	UGCG
Migalastat	PubChem	Approved	UGCG
Miglitol	PubChem	Approved	UGCG