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aceneuramic acid 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |
| Stem definition | Drug id | CAS RN |
|---|---|---|
| 5864 | 131-48-6 |
Description:
| Molecule | Description |
|---|---|
|
Synonyms:
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Aceneuramic acid is a naturally occurring sialic acid that serves as a building block of glycoproteins and glycolipids involved in cell-surface recognition and signaling. It is used as a substrate replacement therapy to increase the availability of sialic acid in patients with GNE myopathy (hereditary inclusion body myopathy), a rare genetic disorder caused by impaired sialic acid biosynthesis due to GNE gene mutations. In Japan, aceneuramic acid has been approved by the PMDA for the treatment of GNE myopathy, with the aim of restoring sialylation of muscle proteins and slowing disease progression.
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Drug dosage:
None
ADMET properties:
None
Predicted pharmacokinetic/toxicological properties (PK-AZ):
None
Approvals:
| Date | Agency | Company | Orphan |
|---|---|---|---|
| March 26, 2024 | PMDA | Nobelpharma Co., Ltd. |
FDA Adverse Event Reporting System (Female)
None
FDA Adverse Event Reporting System (Male)
None
FDA Adverse Event Reporting System (Geriatric)
None
FDA Adverse Event Reporting System (Pediatric)
None
Pharmacologic Action:
| Source | Code | Description |
|---|---|---|
| ATC | M09AX05 | MUSCULO-SKELETAL SYSTEM OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM Other drugs for disorders of the musculo-skeletal system |
| CHEBI has role | CHEBI:22586 | antioxidant |
| CHEBI has role | CHEBI:52425 | EC 3.2.1.18 (<i>exo</i>-α-sialidase) inhibitor |
| CHEBI has role | CHEBI:75771 | mouse metabolite |
| CHEBI has role | CHEBI:76969 | bacterial metabolite |
| CHEBI has role | CHEBI:77746 | human metabolite |
Related Drugs by ATC class:
M09AX Other drugs for disorders of the musculo-skeletal system 11 drugs
Drug Use | Suggest Off label Use Form| |View source of the data|
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Suppression of the progression of muscular weakness in distal myopathy | indication | 702382000 |
🐶 Veterinary Drug Use
None
🐶 Veterinary products
None
Acid dissociation constants calculated using MoKa v3.0.0
None
Orange Book patent data (new drug applications)
None
Orange Book exclusivity data (new drug applications)
None
Bioactivity Summary:
None
External reference:
| ID | Source |
|---|---|
| SIA | PDB_CHEM_ID |
| CHEMBL152452 | ChEMBL_ID |
| CHEMBL2105945 | ChEMBL_ID |
| DB11797 | DRUGBANK_ID |
| C2825327 | UMLSCUI |
| CHEBI:17012 | CHEBI |
| 6697 | INN_ID |
| 445063 | PUBCHEM_CID |
| GZP2782OP0 | UNII |
| 2613558 | RXNORM |
| D11016 | KEGG_DRUG |
| D019158 | MESH_DESCRIPTOR_UI |
| 015301 | NDDF |
Pharmaceutical products:
None