Venglustat
Venglustat is a small molecule pharmaceutical. It is currently being investigated in clinical studies. It is known to target ceramide glucosyltransferase.
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FDA approval date
EMA approval date
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Study first post date
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Completion date
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Commercial
No data
Clinical
Clinical Trials
18 clinical trials
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Indications Phases 4
No data
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Fabry disease | D000795 | Orphanet_324 | E75.21 | — | 2 | 2 | — | — | 4 |
| Gaucher disease | D005776 | Orphanet_355 | E75.22 | — | 1 | 1 | — | — | 2 |
| Polycystic kidney diseases | D007690 | — | Q61.3 | 1 | 1 | 1 | — | — | 2 |
| Autosomal dominant polycystic kidney | D016891 | EFO_1001496 | Q61.2 | 1 | 1 | 1 | — | — | 2 |
| Tay-sachs disease | D013661 | Orphanet_845 | E75.02 | — | — | 1 | — | — | 1 |
| Sandhoff disease | D012497 | Orphanet_796 | E75.01 | — | — | 1 | — | — | 1 |
| Cystic kidney diseases | D052177 | EFO_0008615 | Q61 | — | — | 1 | — | — | 1 |
| Abdominal pain | D015746 | EFO_0003766 | R10.9 | — | — | 1 | — | — | 1 |
Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Parkinson disease | D010300 | EFO_0002508 | G20 | — | 1 | — | — | — | 1 |
| Disease | D004194 | EFO_0000408 | R69 | — | 1 | — | — | — | 1 |
Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Healthy volunteers/patients | — | — | — | 7 | — | — | — | — | 7 |
| Liver diseases | D008107 | EFO_0001421 | K70-K77 | 1 | — | — | — | — | 1 |
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | Venglustat |
| INN | venglustat |
| Description | Venglustat is a small molecule pharmaceutical. It is currently being investigated in clinical studies. It is known to target ceramide glucosyltransferase. |
| Classification | Small molecule |
| Drug class | enzyme inhibitors: glucosyltransferase inhibitors |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | CC(C)(NC(=O)O[C@@H]1CN2CCC1CC2)c1csc(-c2ccc(F)cc2)n1 |
Identifiers
| PDB | — |
| CAS-ID | 1401090-53-6 |
| RxCUI | — |
| ChEMBL ID | CHEMBL4297611 |
| ChEBI ID | — |
| PubChem CID | — |
| DrugBank | — |
| UNII ID | BLP1XA3FZA (ChemIDplus, GSRS) |
Target
Agency Approved
No data
Alternate
UGCG
UGCG
Organism
Homo sapiens
Gene name
UGCG
Gene synonyms
NCBI Gene ID
Protein name
ceramide glucosyltransferase
Protein synonyms
GCS, GLCT-1, Glucosylceramide synthase, Glycosylceramide synthase, UDP-glucose ceramide glucosyltransferase, UDP-glucose:N-acylsphingosine D-glucosyltransferase
Uniprot ID
Mouse ortholog
Ugcg (22234)
ceramide glucosyltransferase (O88693)
Variants
No data
Financial
No data
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 316 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
0 adverse events reported
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