Tradename | Company | Number | Date | Products |
---|---|---|---|---|
ZAVESCA | Actelion | N-021348 RX | 2003-07-31 | 1 products, RLD, RS |
OPFOLDA | Amicus Therapeutics | N-215211 RX | 2023-09-28 | 1 products, RLD, RS |
Indication | Ontology | MeSH | ICD-10 |
---|---|---|---|
gaucher disease | Orphanet_355 | D005776 | E75.22 |
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
---|---|---|---|---|---|---|---|---|---|
Niemann-pick diseases | D009542 | — | E75.24 | — | 1 | — | 1 | — | 2 |
Type a niemann-pick disease | D052536 | EFO_1001380 | E75.240 | — | 1 | — | 1 | — | 2 |
Type c niemann-pick disease | D052556 | Orphanet_646 | E75.242 | — | 1 | — | 1 | — | 2 |
Pick disease of the brain | D020774 | EFO_0003096 | G31.01 | — | — | — | 1 | — | 1 |
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
---|---|---|---|---|---|---|---|---|---|
Fibrosis | D005355 | — | — | — | 3 | 1 | — | — | 3 |
Cystic fibrosis | D003550 | EFO_0000390 | E84 | — | 3 | 1 | — | — | 3 |
Gaucher disease | D005776 | Orphanet_355 | E75.22 | — | — | 1 | — | 1 | 2 |
Tay-sachs disease | D013661 | Orphanet_845 | E75.02 | — | 1 | 1 | — | — | 2 |
Gangliosidoses | D005733 | — | E75.10 | — | 1 | 1 | — | — | 2 |
Gm2 gangliosidoses | D020143 | Orphanet_309152 | E75.0 | — | 1 | 1 | — | — | 2 |
Sandhoff disease | D012497 | Orphanet_796 | E75.01 | — | — | 1 | — | — | 1 |
Drug common name | Miglustat |
INN | miglustat |
Description | Miglustat is a hydroxypiperidine that is deoxynojirimycin in which the amino hydrogen is replaced by a butyl group. It has a role as an EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor and an anti-HIV agent. It is a member of piperidines and a tertiary amino compound. It is functionally related to a duvoglustat. |
Classification | Small molecule |
Drug class | enzyme inhibitors: glucosyltransferase inhibitors |
Image (chem structure or protein) | ![]() |
Structure (InChI/SMILES or Protein Sequence) | CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO |
PDB | — |
CAS-ID | 72599-27-0 |
RxCUI | — |
ChEMBL ID | CHEMBL1029 |
ChEBI ID | 50381 |
PubChem CID | 51634 |
DrugBank | DB00419 |
UNII ID | ADN3S497AZ (ChemIDplus, GSRS) |