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Protein / target

Ceramide glucosyltransferase

Encoded byUGCGQ16739Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
3
Approved medicines
Open Targets target-level
View by indication →
20
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Ceramide glucosyltransferase

Strongest disease association

Gaucher disease

Via encoding gene UGCG · Clinical evidence · score 0.58

Therapeutic position

Established drug target

Small molecules

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function overview

Participates in the initial step of the glucosylceramide-based glycosphingolipid/GSL synthetic pathway at the cytosolic surface of the Golgi.

View complete UniProt function annotation

Participates in the initial step of the glucosylceramide-based glycosphingolipid/GSL synthetic pathway at the cytosolic surface of the Golgi (PubMed:1532799, PubMed:8643456). Catalyzes the transfer of glucose from UDP-glucose to ceramide to produce glucosylceramide/GlcCer (such as beta-D-glucosyl-(1<->1')-N-acylsphing-4-enine) (PubMed:1532799, PubMed:8643456). GlcCer is the core component of glycosphingolipids/GSLs, amphipathic molecules consisting of a ceramide lipid moiety embedded in the outer leaflet of the membrane, linked to one of hundreds of different externally oriented oligosaccharide structures (PubMed:8643456). Glycosphingolipids are essential components of membrane microdomains that mediate membrane trafficking and signal transduction, implicated in many fundamental cellular processes, including growth, differentiation, migration, morphogenesis, cell-to-cell and cell-to-matrix interactions (By similarity). They are required for instance in the proper development and functioning of the nervous system (By similarity). As an example of their role in signal transduction, they regulate the leptin receptor/LEPR in the leptin-mediated signaling pathway (By similarity). They also play an important role in the establishment of the skin barrier regulating keratinocyte differentiation and the proper assembly of the cornified envelope (By similarity). The biosynthesis of GSLs is also required for the proper intestinal endocytic uptake of nutritional lipids (By similarity). Catalyzes the synthesis of xylosylceramide/XylCer (such as beta-D-xylosyl-(1<->1')-N-acylsphing-4-enine) using UDP-Xyl as xylose donor (PubMed:33361282)

Subcellular location

Golgi apparatus membrane
Domains and Gene Ontology detail (15)

Gene Ontology

  • CGolgi membrane
  • Cmembrane
  • Fceramide glucosyltransferase activity
  • Pcell differentiation
  • Pcornified envelope assembly
  • Pepidermis development
  • Pestablishment of skin barrier
  • Pglucosylceramide biosynthetic process
  • Pglycosphingolipid biosynthetic process
  • Pintestinal lipid absorption
  • Pkeratinocyte differentiation
  • Pleptin-mediated signaling pathway

394 aa · 45 kDa

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

View underlying pathways (1)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

GBAB4GALT6CERKGBA2SMPD2CERS6DEGS1ASAH1CERS2SMPD1UGCG

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Approved medicines with mapped indications

1 medicine · 3 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Gaucher disease1 medicine
Glycogen Storage Disease Type II1 medicine
Niemann-Pick Diseases1 medicine

3 medicines meet Open Targets' target-level approved-medicine definition; the 1 shown here are those on this page with a canonical approved disease indication in the graph. Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

1

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

miglustat
Narrow target profileApprovedInhibitor

Ceramide glucosyltransferase inhibitor

Indicated for Gaucher disease, Glycogen Storage Disease Type II, Niemann-Pick Diseases

Direct interaction with this protein · Only this protein recorded as a target

ChEMBL mechanism, action type, target identity and approved indications. Open Targets clinical status.

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene UGCG

Gene-level evidence surfaced through the gene UGCGthat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Gaucher disease
0.72Moderately supported

Clinical evidence dominant · Open Targets 0.58

Gaucher disease type 1
0.62Moderately supported

Clinical evidence dominant · Open Targets 0.50

Niemann-Pick Disease, Type C
0.62Moderately supported

Clinical evidence dominant · Open Targets 0.50

Fabry Disease
0.49Limited support

Clinical evidence dominant · Open Targets 0.39

Metabolic Diseases
0.46Limited support

Clinical evidence dominant · Open Targets 0.37

View evidence synthesis (5)
Gaucher diseaseModerately supported
0.72
agreement 0.560.87
Clinical96%Literature4%

Open Targets aggregate 0.58 · 2 independent evidence families

Gaucher disease type 1Moderately supported
0.62
agreement 0.470.78
Clinical97%Literature3%

Open Targets aggregate 0.50 · 2 independent evidence families

Niemann-Pick Disease, Type CModerately supported
0.62
agreement 0.460.77
Clinical98%Literature2%

Open Targets aggregate 0.50 · 2 independent evidence families

Fabry DiseaseLimited support
0.49
agreement 0.330.64
Clinical95%Literature5%

Open Targets aggregate 0.39 · 2 independent evidence families

Metabolic DiseasesLimited support
0.46
agreement 0.300.61
Clinical99%Literature1%

Open Targets aggregate 0.37 · 2 independent evidence families

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.

Show all associations
Gaucher disease0.58
Gaucher disease type 10.50
Niemann-Pick Disease, Type C0.50
Fabry Disease0.39
Neurodegenerative Diseases0.37
Metabolic Diseases0.37

Drug development

5 compounds recorded · 3 approved · 2 in clinical development

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 1 drug that targets this protein in Forefront's canonical graph (1 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (5)
VENGLUSTATPhase 3
LUCERASTATPhase 3
ELIGLUSTAT TARTRATEApproval
MIGLUSTATApproval
ELIGLUSTATApproval

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small moleculesStrong

Approved Drug and High-Quality Ligand support this modality.

AntibodiesEmerging

Feasibility evidence (uniprot sigp or tmhmm) — no clinical-stage drug of this modality recorded.

Protein degradersEmerging

Feasibility evidence (database ubiquitination and half-life data) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (7)
SM · Approved DrugSM · High-Quality LigandSM · Druggable FamilyAB · UniProt SigP or TMHMMPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Clinical trials

20

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

View all trials (16)

COMPLETED · via miglustat · NCT00194649

COMPLETED · via miglustat · NCT01760564

ClinicalTrials.gov via the drug-target graph.

What's happening now

5

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for a drug that targets this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  1. Regulatory approval2023-06-26

    Approval: Opfolda (EMA)

    ema · regulatory · ema · via miglustat

  2. Regulatory approval2019-02-18

    Approval: Miglustat Dipharma (EMA)

    ema · regulatory · ema · via miglustat

  3. Regulatory approval2017-11-09

    Approval: Miglustat Gen.Orph (EMA)

    ema · regulatory · ema · via miglustat

  4. Regulatory approval2017-03-22

    Approval: Yargesa (EMA)

    ema · regulatory · ema · via miglustat

  5. Regulatory approval2002-11-20

    Approval: Zavesca (EMA)

    ema · regulatory · ema · via miglustat

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.