This brand name is authorized in France, Lithuania, Romania.
The drug WAINZUA contains one active pharmaceutical ingredient (API):
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UNII
WSP2DHR2BD - EPLONTERSEN SODIUM
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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. |
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Below package inserts are available for further reading:
| Document | Type | Information Source | |
|---|---|---|---|
| WAINZUA Solution for injection | MPI, EU: SmPC | European Medicines Agency (EU) |
This drug has been classified in the anatomical therapeutic chemical (ATC) classification system as follows:
| ATC Group | Classification | |
|---|---|---|
| N07XX21 | N Nervous system → N07 Other nervous system drugs → N07X Other nervous system drugs → N07XX Other nervous system drugs | |
This drug has been assigned below unique identifiers within the countries it is being marketed:
| Country | Identification scheme | Identifier(s) |
|---|---|---|
| FR | Base de données publique des médicaments | 65079351 |
| LT | Valstybinė vaistų kontrolės tarnyba | 1103018 |
| RO | Agenția Națională a Medicamentului și a Dispozitivelor Medicale | W70969001 |
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