Stem definition | Drug id | CAS RN |
---|---|---|
enzyme inhibitors | 1807 | 72599-27-0 |
Dose | Unit | Route |
---|---|---|
0.30 | g | O |
Property | Value | Reference |
---|---|---|
BDDCS (Biopharmaceutical Drug Disposition Classification System) | 3 | Benet LZ, Broccatelli F, Oprea TI |
S (Water solubility) | 1000 mg/mL | Benet LZ, Broccatelli F, Oprea TI |
EoM (Fraction excreted unchanged in urine) | 85 % | Benet LZ, Broccatelli F, Oprea TI |
MRTD (Maximum Recommended Therapeutic Daily Dose) | 19.54 µM/kg/day | Contrera JF, Matthews EJ, Kruhlak NL, Benz RD |
BA (Bioavailability) | 97 % | Kim MT, Sedykh A, Chakravarti SK, Saiakhov RD, Zhu H |
Date | Agency | Company | Orphan |
---|---|---|---|
Nov. 20, 2002 | EMA | ||
July 31, 2003 | FDA | ACTELION PHARMS LTD |
MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
---|---|---|---|---|---|---|
Niemann-Pick disease | 421.06 | 39.50 | 44 | 1184 | 6 | 53347832 |
Seizure | 139.92 | 39.50 | 65 | 1163 | 129444 | 53218394 |
Dysphagia | 68.59 | 39.50 | 35 | 1193 | 84362 | 53263476 |
Gastrostomy | 62.58 | 39.50 | 11 | 1217 | 587 | 53347251 |
Epilepsy | 61.93 | 39.50 | 23 | 1205 | 25744 | 53322094 |
Disease progression | 59.66 | 39.50 | 34 | 1194 | 101886 | 53245952 |
Tremor | 58.10 | 39.50 | 36 | 1192 | 125700 | 53222138 |
Diarrhoea | 56.39 | 39.50 | 70 | 1158 | 625476 | 52722362 |
Weight decreased | 47.98 | 39.50 | 41 | 1187 | 234907 | 53112931 |
Pneumonia aspiration | 45.58 | 39.50 | 20 | 1208 | 34281 | 53313557 |
Gastrointestinal tube insertion | 43.39 | 39.50 | 9 | 1219 | 1158 | 53346680 |
Concomitant disease aggravated | 41.46 | 39.50 | 13 | 1215 | 8725 | 53339113 |
MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
---|---|---|---|---|---|---|
Niemann-Pick disease | 368.85 | 41.78 | 42 | 1165 | 23 | 32512296 |
Seizure | 121.19 | 41.78 | 64 | 1143 | 103790 | 32408529 |
Gastrostomy | 78.78 | 41.78 | 15 | 1192 | 770 | 32511549 |
Concomitant disease aggravated | 58.03 | 41.78 | 17 | 1190 | 5636 | 32506683 |
Disease progression | 49.71 | 41.78 | 34 | 1173 | 86828 | 32425491 |
Diarrhoea | 48.43 | 41.78 | 63 | 1144 | 364739 | 32147580 |
Pneumonia aspiration | 46.63 | 41.78 | 25 | 1182 | 41131 | 32471188 |
Source | Code | Description |
---|---|---|
ATC | A16AX06 | ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Various alimentary tract and metabolism products |
FDA MoA | N0000020019 | Glucosylceramide Synthase Inhibitors |
MeSH PA | D019380 | Anti-HIV Agents |
MeSH PA | D000890 | Anti-Infective Agents |
MeSH PA | D044966 | Anti-Retroviral Agents |
MeSH PA | D000998 | Antiviral Agents |
MeSH PA | D004791 | Enzyme Inhibitors |
MeSH PA | D065089 | Glycoside Hydrolase Inhibitors |
MeSH PA | D007004 | Hypoglycemic Agents |
FDA EPC | N0000175783 | Glucosylceramide Synthase Inhibitor |
CHEBI has role | CHEBI:50382 | glucosylceramide synthase inhibitors |
CHEBI has role | CHEBI:64946 | anti-HIV agents |
Disease | Relation | SNOMED_ID | DOID |
---|---|---|---|
Niemann-Pick disease, type C | indication | 66751000 | |
Glucosylceramide beta-glucosidase deficiency | indication | 190794006 | DOID:1926 |
Impaired renal function disorder | contraindication | 197663003 | |
Pregnancy, function | contraindication | 289908002 | |
Peripheral nerve disease | contraindication | 302226006 | |
Breastfeeding (mother) | contraindication | 413712001 |
Dissociation level | Dissociation constant | Type (acidic/basic) |
---|---|---|
pKa1 | 13.42 | acidic |
pKa2 | 7.76 | Basic |
None
None
Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
---|---|---|---|---|---|---|---|---|---|
Ceramide glucosyltransferase | Enzyme | INHIBITOR | Ki | 5.13 | IUPHAR | CHEMBL | |||
Lysosomal alpha-glucosidase | Enzyme | IC50 | 7 | CHEMBL | |||||
Non-lysosomal glucosylceramidase | Enzyme | IC50 | 6.64 | CHEMBL | |||||
Sucrase-isomaltase, intestinal | Enzyme | IC50 | 6.30 | CHEMBL | |||||
Glycogen debranching enzyme | Enzyme | IC50 | 5 | CHEMBL | |||||
Maltase-glucoamylase, intestinal | Enzyme | IC50 | 5.68 | CHEMBL | |||||
Sucrase-isomaltase, intestinal | Enzyme | IC50 | 6.24 | CHEMBL | |||||
Non-lysosomal glucosylceramidase | Enzyme | IC50 | 6.85 | CHEMBL | |||||
Lysosomal alpha-glucosidase | Enzyme | IC50 | 5.68 | CHEMBL | |||||
Lysosomal alpha-glucosidase | Enzyme | IC50 | 5.05 | CHEMBL | |||||
Sucrase-isomaltase | Enzyme | IC50 | 6.22 | CHEMBL | |||||
Putative alpha-glucosidase | Enzyme | IC50 | 6.38 | CHEMBL | |||||
Trehalase | Enzyme | IC50 | 4.89 | CHEMBL | |||||
Uncharacterized protein | Enzyme | IC50 | 4.44 | CHEMBL | |||||
Ceramide glucosyltransferase | Enzyme | IC50 | 4.64 | CHEMBL |
ID | Source |
---|---|
4021406 | VUID |
N0000148822 | NUI |
D05032 | KEGG_DRUG |
4021406 | VANDF |
C1321596 | UMLSCUI |
CHEBI:50381 | CHEBI |
NBV | PDB_CHEM_ID |
CHEMBL1029 | ChEMBL_ID |
DB00419 | DRUGBANK_ID |
51634 | PUBCHEM_CID |
8138 | INN_ID |
C059896 | MESH_SUPPLEMENTAL_RECORD_UI |
4841 | IUPHAR_LIGAND_ID |
ADN3S497AZ | UNII |
356766 | RXNORM |
17531 | MMSL |
276708 | MMSL |
47238 | MMSL |
d04889 | MMSL |
009940 | NDDF |
408044003 | SNOMEDCT_US |
412296005 | SNOMEDCT_US |
Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
---|---|---|---|---|---|---|---|---|
Miglustat | HUMAN PRESCRIPTION DRUG LABEL | 1 | 10148-201 | CAPSULE | 100 mg | ORAL | NDA AUTHORIZED GENERIC | 29 sections |
Miglustat | Human Prescription Drug Label | 1 | 42799-708 | CAPSULE | 100 mg | ORAL | ANDA | 23 sections |
MIGLUSTAT | HUMAN PRESCRIPTION DRUG LABEL | 1 | 43975-310 | CAPSULE | 100 mg | ORAL | ANDA | 26 sections |
Zavesca | HUMAN PRESCRIPTION DRUG LABEL | 1 | 66215-201 | CAPSULE | 100 mg | ORAL | NDA | 29 sections |