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AboutPricing
Drug ReportsMiglustat
Zavesca(miglustat)
Miglustat, Opfolda, Yargesa, 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, glycogen storage disease type II, and niemann-pick diseases. The pharmaceutical is active against ceramide glucosyltransferase.
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Commercial
Clinical
Drug
Target
Variants
Financial
Trends
Safety
Events Timeline
5D
1M
3M
6M
YTD
1Y
2Y
5Y
Max
Events
FDA approval date
EMA approval date
Patent expiration date
Study first post date
Last update post date
Start date
Primary completion date
Completion date
Results first post date
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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
Miglustat, Opfolda, Yargesa, Zavesca (discontinued: Miglustat)
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
OPFOLDAAmicus TherapeuticsN-215211 RX2023-09-28
1 products, RLD, RS
Labels
FDA
EMA
Brand Name
Status
Last Update
miglustatANDA2025-08-07
opfoldaNew Drug Application2024-11-27
yargesaANDA2025-04-09
zavecaNew Drug Application2010-05-21
zavescaNew Drug Application2023-04-20
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
31 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
Glycogen storage disease type iiD006009Orphanet_365E74.02214—28
Indications Phases 2
No data
Indications Phases 1
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
HypersensitivityD006967EFO_0003785T78.401————1
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)Loading
Structure (InChI/SMILES or Protein Sequence)
CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO
Identifiers
PDB—
CAS-ID72599-27-0
RxCUI—
ChEMBL IDCHEMBL1029
ChEBI ID50381
PubChem CID51634
DrugBankDB00419
UNII IDADN3S497AZ (ChemIDplus, GSRS)
Target
Agency Approved
No data
Alternate
GBA1
GBA1
Organism
Homo sapiens
Gene name
GBA1
Gene synonyms
GBA, GC, GLUC
NCBI Gene ID
2629
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
Q9UMJ8
Mouse ortholog
Gba1 (14466)
lysosomal acid glucosylceramidase (Q78NR7)
Variants
No data
Financial
No data
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 2,284 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
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1,271 adverse events reported
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