MIGLUSTAT

Search structure
Trade Names:
Synonyms:
Status: Approved (2003)
Entry Type: Small molecule
Molecule Category: UNKNOWN
ATC: A16AX06
UNII: ADN3S497AZ

Structure
InChI Key UQRORFVVSGFNRO-UTINFBMNSA-N
Smile CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO
InChI
InChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9-,10-/m1/s1

Physicochemical Descriptors

Property Name Value
Molecular Formula C10H21NO4
Molecular Weight 219.28
AlogP -1.45
Hydrogen Bond Acceptor 5.0
Hydrogen Bond Donor 4.0
Number of Rotational Bond 4.0
Polar Surface Area 84.16
Molecular species NEUTRAL
Aromatic Rings 0.0
Heavy Atoms 15.0

Pharmacology

Action Mechanism of Action Reference
INHIBITOR Ceramide glucosyltransferase inhibitor DailyMed
Primary Target
UDP-glucose ceramide glucosyltransferase
Targets EC50(nM) IC50(nM) Kd(nM) Ki(nM) Inhibition(%)
Enzyme Hydrolase
- 100-100 - - -
Enzyme Transferase
- 20000-50000 - - -
Enzyme
- 14-300 - - 70

Indications

Mesh Heading Maximum Phase Mesh ID Reference
Gaucher Disease 4 D005776 ClinicalTrials
Glycogen Storage Disease Type II 3 D006009 ClinicalTrials
Sandhoff Disease 3 D012497 ClinicalTrials
Gangliosidoses, GM2 3 D020143 ClinicalTrials
Glycogen Storage Disease 3 D006008 ClinicalTrials
Niemann-Pick Disease, Type C 3 D052556 ClinicalTrials
HIV Infections 2 D015658 ClinicalTrials
Spastic Paraplegia, Hereditary 2 D015419 ClinicalTrials
Cystic Fibrosis 2 D003550 ClinicalTrials

Related Entries

MCS

Side Effects from Label

Side effects Relative Frequency (%) Labels
Gastrointestinal disorders Diarrhoea 100.0
Gastrointestinal disorders Diarrhoea 89.0
Gastrointestinal disorders Diarrhoea 83.0
Gastrointestinal disorders Abdominal pain 67.0
Gastrointestinal disorders Gastrointestinal pain 67.0
Investigations Weight decreased 67.0
Investigations Weight decreased 63.7
Gastrointestinal disorders Abdominal pain 58.0
Gastrointestinal disorders Gastrointestinal pain 58.0
Gastrointestinal disorders Abdominal pain 50.0
Gastrointestinal disorders Flatulence 50.0
Gastrointestinal disorders Gastrointestinal pain 50.0
Gastrointestinal disorders Flatulence 45.0
Gastrointestinal disorders Flatulence 44.0
Gastrointestinal disorders Abdominal pain 42.5
Gastrointestinal disorders Gastrointestinal pain 42.5
Gastrointestinal disorders Flatulence 42.0
Investigations Weight decreased 42.0
Investigations Weight decreased 39.0
Nervous system disorders Tremor 33.0
Gastrointestinal disorders Flatulence 29.0
Gastrointestinal disorders Constipation 25.0
Cardiac disorders Dizziness 25.0
Nervous system disorders Tremor 22.5
Nervous system disorders Headache 22.0
Gastrointestinal disorders Nausea 22.0
Nervous system disorders Headache 21.0
Gastrointestinal disorders Abdominal pain 18.0
Gastrointestinal disorders Gastrointestinal pain 18.0
General disorders and administration site conditions Asthenia 17.0
Nervous system disorders Tremor 17.0
Eye disorders Visual impairment 17.0
Nervous system disorders Headache 15.0
Gastrointestinal disorders Nausea 14.0
Gastrointestinal disorders Nausea 11.2
Cardiac disorders Dizziness 11.0
Musculoskeletal and connective tissue disorders Muscle spasms 11.0
Nervous system disorders Tremor 11.0
Gastrointestinal disorders Vomiting 11.0
Cardiac disorders Dizziness 10.0
Gastrointestinal disorders Constipation 8.75
Gastrointestinal disorders Abdominal distension 8.0
Nervous system disorders Amnesia 8.0
General disorders and administration site conditions Asthenia 8.0
Musculoskeletal and connective tissue disorders Back pain 8.0
General disorders and administration site conditions Chills 8.0
Congenital, familial and genetic disorders Congenital eye disorder 8.0
Gastrointestinal disorders Constipation 8.0
General disorders and administration site conditions Decreased appetite 8.0
Cardiac disorders Dizziness 8.0
Gastrointestinal disorders Dry mouth 8.0
Eye disorders Eye disorder 8.0
General disorders and administration site conditions Feeling jittery 8.0
General disorders and administration site conditions Gait disturbance 8.0
Nervous system disorders Hypoaesthesia 8.0
General disorders and administration site conditions Influenza like illness 8.0
Endocrine disorders Menstruation irregular 8.0
Musculoskeletal and connective tissue disorders Muscle spasms 8.0
Gastrointestinal disorders Nausea 8.0
Psychiatric disorders Nervousness 8.0
General disorders and administration site conditions Pain 8.0
Musculoskeletal and connective tissue disorders Pain in extremity 8.0
Sensation of heaviness 8.0
Nervous system disorders Tremor 8.0
Eye disorders Visual impairment 8.0
Nervous system disorders Paraesthesia 7.5
General disorders and administration site conditions Decreased appetite 7.0
Gastrointestinal disorders Dyspepsia 7.0
Nervous system disorders Paraesthesia 7.0
Blood and lymphatic system disorders Thrombocytopenia 7.0
Gastrointestinal disorders Abdominal distension 6.0
Gastrointestinal disorders Abdominal pain upper 6.0
Gastrointestinal disorders Flatulence 6.0
Reproductive system and breast disorders Menstrual disorder 6.0
Nervous system disorders Migraine 6.0
Blood and lymphatic system disorders Thrombocytopenia 6.0
General disorders and administration site conditions Asthenia 5.0
General disorders and administration site conditions Decreased appetite 5.0
Blood and lymphatic system disorders Thrombocytopenia 5.0
Musculoskeletal and connective tissue disorders Muscle spasms 4.0
Gastrointestinal disorders Vomiting 4.0
Nervous system disorders Neuropathy peripheral 3.75
Gastrointestinal disorders Vomiting 3.75
Gastrointestinal disorders Abdominal distension 2.5
General disorders and administration site conditions Asthenia 2.5
General disorders and administration site conditions Decreased appetite 2.5
Gastrointestinal disorders Dry mouth 2.5
Gastrointestinal disorders Dyspepsia 2.5
General disorders and administration site conditions Fatigue 2.5
General disorders and administration site conditions Influenza like illness 2.5
Ear and labyrinth disorders Vertigo 2.5

Adverse Reactions

SOC By Demographic Distribution
System Organ Classification (SOC)
Relative Frequency (%)
Nervous system disorders
16.32
Gastrointestinal disorders
12.7
General disorders and administration site conditions
11.16
Respiratory, thoracic and mediastinal disorders
10.27
Investigations
7.14
Infections and infestations
6.88
Psychiatric disorders
5.87
Metabolism and nutrition disorders
4.6
Surgical and medical procedures
3.74
Musculoskeletal and connective tissue disorders
3.01
Vascular disorders
2.8
Injury, poisoning and procedural complications
2.26
Blood and lymphatic system disorders
2.21
Congenital, familial and genetic disorders
2.11

Cross References

Resources Reference
CAS NUMBER 72599-27-0
ChEBI 50381
ChEMBL CHEMBL1029
DrugBank DB00419
DrugCentral 1807
EPA CompTox DTXSID6045618
FDA SRS ADN3S497AZ
Human Metabolome Database HMDB0014563
Guide to Pharmacology 4841
KEGG D05032
PDB NBV
PharmGKB PA10140
PubChem 51634
SureChEMBL SCHEMBL246893
ZINC ZINC000003794711
CONTENTS