| Stem definition | Drug id | CAS RN |
|---|---|---|
| antisense oligonucleotides | 5335 | 915430-78-3 |
| Molecule | Description |
|---|---|
|
Synonyms:
|
Volanesorsen is an antisense oligonucleotide designed to inhibit the formation of apoC-III, a protein that is recognised to regulate both triglyceride metabolism and hepatic clearance of chylomicrons and other triglyceride-rich lipoproteins. The selective binding of volanesorsen to the apoC-III messenger ribonucleic acid (mRNA) within the 3′ untranslated region at base position 489-508 causes the degradation of the mRNA. This binding prevents translation of the protein apoC-III, thus removing an inhibitor of triglyceride clearance and enabling metabolism through an LPL-independent pathway
|
| Dose | Unit | Route |
|---|---|---|
| 20 | mg | P |
None
| Date | Agency | Company | Orphan |
|---|---|---|---|
| May 3, 2019 | EMA | Akcea Therapeutics Ireland Limited |
None
None
None
None
| Source | Code | Description |
|---|---|---|
| ATC | C10AX18 | CARDIOVASCULAR SYSTEM LIPID MODIFYING AGENTS LIPID MODIFYING AGENTS, PLAIN Other lipid modifying agents |
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Chylomicronemia syndrome | indication | 402786009 | |
| Thrombocytopenic disorder | contraindication | 302215000 | DOID:1588 |
None
None
None
None
None
| Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
|---|---|---|---|---|---|---|---|---|---|
| apolipoprotein C-III (apoC-III) mRNA | RNA | ANTISENSE INHIBITOR | UNKNOWN | DRUG LABEL |
| ID | Source |
|---|---|
| 2O4BE0K238 | UNII |
| 1573402-50-2 | SECONDARY_CAS_RN |
| C4726970 | UMLSCUI |
| CHEMBL3544994 | ChEMBL_ID |
| 121494123 | PUBCHEM_CID |
| DB15067 | DRUGBANK_ID |
| CHEMBL3544989 | ChEMBL_ID |
| 10166 | INN_ID |
| 8681 | IUPHAR_LIGAND_ID |
| 018803 | NDDF |
| 018804 | NDDF |
| 2637345 | RXNORM |
| 37541 | MMSL |
| C000593612 | MESH_SUPPLEMENTAL_RECORD_UI |
None