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
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 |
|---|---|---|---|---|
| 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 |
Indications
FDA
EMA
Indication | Ontology | MeSH | ICD-10 |
|---|---|---|---|
| gaucher disease | Orphanet_355 | D005776 | E75.22 |
Agency Specific
FDA
EMA
No data
Patent Expiration
No data
HCPCS
No data
Clinical
Clinical Trials
31 clinical trials
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Indications Phases 4
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Niemann-pick diseases | D009542 | — | E75.24 | 1 | 1 | 1 | 1 | — | 4 |
| Type a niemann-pick disease | D052536 | EFO_1001380 | E75.240 | 1 | 1 | 1 | 1 | — | 4 |
| Type c niemann-pick disease | D052556 | Orphanet_646 | E75.242 | 1 | 1 | 1 | 1 | — | 4 |
| Tay-sachs disease | D013661 | Orphanet_845 | E75.02 | — | 1 | 2 | 1 | — | 4 |
| Gangliosidoses | D005733 | — | E75.10 | — | 1 | 2 | 1 | — | 4 |
| Gm2 gangliosidoses | D020143 | Orphanet_309152 | E75.0 | — | 1 | 2 | 1 | — | 4 |
| Pick disease of the brain | D020774 | EFO_0003096 | G31.01 | 1 | — | 1 | 1 | — | 3 |
| Sandhoff disease | D012497 | Orphanet_796 | E75.01 | — | — | 1 | 1 | — | 2 |
| Gm1 gangliosidosis | D016537 | Orphanet_354 | E75.19 | — | — | — | 1 | — | 1 |
| Contraception | D003267 | — | — | — | — | — | 1 | — | 1 |
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Glycogen storage disease type ii | D006009 | Orphanet_365 | E74.02 | 2 | 1 | 4 | — | 3 | 9 |
| Fibrosis | D005355 | — | — | — | 4 | 1 | — | — | 4 |
| Cystic fibrosis | D003550 | EFO_0000390 | E84 | — | 4 | 1 | — | — | 4 |
| Gaucher disease | D005776 | Orphanet_355 | E75.22 | — | 1 | 1 | — | 1 | 3 |
| Disease | D004194 | EFO_0000408 | R69 | — | — | 1 | — | — | 1 |
| Frontotemporal dementia | D057180 | HP_0002145 | G31.0 | — | — | 1 | — | — | 1 |
| Primary progressive aphasia | D018888 | EFO_0009053 | G31.01 | — | — | 1 | — | — | 1 |
| Glycogen storage disease | D006008 | — | E74.0 | — | — | 1 | — | — | 1 |
Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Hiv infections | D015658 | EFO_0000764 | B20 | 1 | 2 | — | — | — | 3 |
| Muscle spasticity | D009128 | HP_0001257 | — | — | 1 | — | — | — | 1 |
| Hereditary spastic paraplegia | D015419 | EFO_0000529 | G11.4 | — | 1 | — | — | — | 1 |
| Paraplegia | D010264 | HP_0001258 | G82.2 | — | 1 | — | — | — | 1 |
| Paraparesis | D020335 | HP_0002385 | G82.2 | — | 1 | — | — | — | 1 |
| Spastic paraparesis | D020336 | HP_0002313 | — | — | 1 | — | — | — | 1 |
| Neuronal ceroid-lipofuscinoses | D009472 | — | E75.4 | 1 | 1 | — | — | — | 1 |
Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Diarrhea | D003967 | HP_0002014 | R19.7 | 1 | — | — | — | — | 1 |
| Hypersensitivity | D006967 | HP_0012393 | T78.40 | 1 | — | — | — | — | 1 |
| Acquired immunodeficiency syndrome | D000163 | EFO_0000765 | B20 | 1 | — | — | — | — | 1 |
| Immunologic deficiency syndromes | D007153 | HP_0002721 | D84.9 | 1 | — | — | — | — | 1 |
| Aids-related complex | D000386 | EFO_0007137 | B20 | 1 | — | — | — | — | 1 |
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| 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 |
Identifiers
| PDB | — |
| CAS-ID | 72599-27-0 |
| RxCUI | — |
| ChEMBL ID | CHEMBL1029 |
| ChEBI ID | 50381 |
| PubChem CID | 51634 |
| DrugBank | DB00419 |
| UNII ID | ADN3S497AZ (ChemIDplus, GSRS) |
Target
Agency Approved
No data
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
No data
Financial
No data
Trends
PubMed Central
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Safety
Black-box Warning
No Black-box warning
Adverse Events
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1,194 adverse events reported
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