The World Health Organization's ATC classification organizes medical drugs based on therapeutic properties, chemical composition, and anatomy. It helps make essential medicines readily available globally and is widely used in the pharmaceutical industry.
| Level | Code | Title | |
|---|---|---|---|
1
|
N | Nervous system | |
2
|
N07 | Other nervous system drugs | |
3
|
N07X | Other nervous system drugs | |
4
|
N07XX | Other nervous system drugs | |
5
|
N07XX21 |
| Active Ingredient |
|---|
|
Eplontersen is a N-acetylgalactosamine (GalNAc)-conjugated 2′-O-2-methoxyethyl-modified chimeric gapmer antisense oligonucleotide (ASO) with a mixed backbone of phosphorothioate and phosphate diester internucleotide linkages. The GalNAc conjugate enables targeted delivery of the ASO to hepatocytes. The selective binding of eplontersen to the transthyretin (TTR) messenger RNA (mRNA) within the hepatocytes causes the degradation of both mutant and wild type (normal) TTR mRNA. This prevents the synthesis of TTR protein in the liver, resulting in significant reductions in the levels of mutated and wild type TTR protein secreted by the liver into the circulation. |
| Document | Type | Information Source | |
|---|---|---|---|
| WAINUA Solution for injection | MPI, US: SPL/PLR | FDA, National Drug Code (US) | |
| WAINZUA Solution for injection | MPI, EU: SmPC | European Medicines Agency (EU) |
Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide.