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Zavesca(miglustat)
Zavesca (miglustat) is a small molecule pharmaceutical. Miglustat was first approved as Zavesca on 2002-11-20. It is used to treat gaucher disease in the USA. It has been approved in Europe to treat gaucher disease and niemann-pick diseases. The pharmaceutical is active against ceramide glucosyltransferase. In addition, it is known to target lysosomal acid glucosylceramidase.
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Commercial
Therapeutic Areas
Therapeutic Area
MeSH
nervous system diseasesD009422
hemic and lymphatic diseasesD006425
hereditary congenital and neonatal diseases and abnormalitiesD009358
nutritional and metabolic diseasesD009750
Trade Name
FDA
EMA
Zavesca (generic drugs available since 2018-04-17)
Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
Miglustat
Tradename
Company
Number
Date
Products
ZAVESCAActelionN-021348 RX2003-07-31
1 products, RLD, RS
Labels
FDA
EMA
Brand Name
Status
Last Update
miglustatNDA authorized generic2023-04-25
zavescaNew Drug Application2021-01-20
Indications
FDA
EMA
Indication
Ontology
MeSH
ICD-10
gaucher diseaseOrphanet_355D005776E75.22
Agency Specific
FDA
EMA
No data
Patent Expiration
No data
ATC Codes
A: Alimentary tract and metabolism drugs
A16: Other alimentary tract and metabolism products in atc
A16A: Other alimentary tract and metabolism products in atc
A16AX: Various alimentary tract and metabolism products
A16AX06: Miglustat
HCPCS
No data
Clinical
Clinical Trials
25 clinical trials
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Mock data
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Indications Phases 4
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Gm2 gangliosidosesD020143Orphanet_309152E75.01214
Type c niemann-pick diseaseD052556Orphanet_646E75.2421113
Tay-sachs diseaseD013661Orphanet_845E75.02112
Sandhoff diseaseD012497Orphanet_796E75.01112
ContraceptionD00326711
Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Glycogen storage disease type iiD006009Orphanet_365E74.021214
Cystic fibrosisD003550EFO_0000390E84414
Gaucher diseaseD005776Orphanet_355E75.221113
Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Hiv infectionsD015658EFO_0000764B20123
Hereditary spastic paraplegiaD015419EFO_0000529G11.411
Neuronal ceroid-lipofuscinosesD009472E75.4111
Indications Phases 1
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
HypersensitivityD006967EFO_0003785T78.4011
Niemann-pick diseasesD009542E75.2411
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameMIGLUSTAT
INNmiglustat
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 classenzyme 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
Identifiers
PDB2V3D
CAS-ID72599-27-0
RxCUI402316
ChEMBL IDCHEMBL1029
ChEBI ID50381
PubChem CID51634
DrugBankDB00419
UNII IDADN3S497AZ (ChemIDplus, GSRS)
Target
Agency Approved
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)
Alternate
GBA1
GBA1
Organism
Homo sapiens
Gene name
GBA1
Gene synonyms
GBA, GC, GLUC
NCBI Gene ID
Protein name
lysosomal acid glucosylceramidase
Protein synonyms
Acid beta-glucosidase, Alglucerase, Beta-GC, Beta-glucocerebrosidase, Beta-glucosylceramidase 1, Cholesterol glucosyltransferase, Cholesteryl-beta-glucosidase, D-glucosyl-N-acylsphingosine glucohydrolase, glucocerebrosidase, glucosidase, beta, acid, Glucosylceramidase beta 1, glucosylceramidase-like protein, Imiglucerase, lysosomal acid GCase, Lysosomal cholesterol glycosyltransferase, Lysosomal galactosylceramidase, lysosomal glucocerebrosidase, Lysosomal glycosylceramidase, SGTase
Uniprot ID
Mouse ortholog
Gba1 (14466)
lysosomal acid glucosylceramidase (Q78NR7)
Variants
Clinical Variant
No data
Financial
No data
Trends
PubMed Central
Top Terms for Disease or Syndrome:
Mock data
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Additional graphs summarizing 1,487 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
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81 adverse events reported
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