Eplontersen
Antisense Oligonucleotide — NERVOUS SYSTEM
Also known as: AKCEA-TTR-LRx, eplontersen sodium, ION-682884, IONIS-TTR-LRx, wainua
Data last refreshed: (DrugCentral release 2024-11-01)
Key facts
Eplontersen is a drug in the Antisense Oligonucleotide class catalogued in DrugCentral. It was first approved by the FDA in 2023. DrugCentral lists 1 molecular target, including TTR. Recorded indications include Polyneuropathy of hereditary transthyretin-mediated amyloidosis.
- Drug class: Antisense Oligonucleotide; Transthyretin-directed RNA Interaction.
- Molecular targets: TTR (1 listed in DrugCentral).
- Indications: Polyneuropathy of hereditary transthyretin-mediated amyloidosis.
- ATC code: N07XX21 (Other nervous system drugs).
Indications & contraindications
Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).
Indications (1)
- Polyneuropathy of hereditary transthyretin-mediated amyloidosis
Mechanism & targets
| Established pharmacologic class | Antisense Oligonucleotide; Transthyretin-directed RNA Interaction |
|---|
Molecular targets (1)
| Target | Gene | Action | Activity |
|---|---|---|---|
| transthyretin (TTR) mRNA | TTR | other | IC50 7.23 |
Regulatory approvals (1)
| Date | Agency | Marketed as | Applicant | Orphan |
|---|---|---|---|---|
| 2023-12-21 | FDA | — | IONIS PHARMS INC | Yes |
ATC classification
| ATC code | Therapeutic hierarchy |
|---|---|
N07XX21 | NERVOUS SYSTEM › OTHER NERVOUS SYSTEM DRUGS › OTHER NERVOUS SYSTEM DRUGS › Other nervous system drugs |
Pharmacogenomics
Drug-label and guideline annotations from PharmGKB and CPIC.
- EMA label: TTR
- FDA label: TTR · Testing Required
- HCSC label: TTR
Chemical structure
| Molecular formula | — |
|---|---|
| Molecular weight | — |
| CAS number | — |
| InChIKey | — |
| PubChem CID | — |
Frequently asked questions
Answers are generated from the structured data on this page, not written by hand.
What is Eplontersen used for?
DrugCentral lists the following indications for Eplontersen: Polyneuropathy of hereditary transthyretin-mediated amyloidosis.
How does Eplontersen work?
Its molecular targets in DrugCentral include TTR.
When was Eplontersen first approved?
Eplontersen was first approved by the FDA in 2023.
What drug class does Eplontersen belong to?
Eplontersen is in the Antisense Oligonucleotide class.
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Sources
- DrugCentral drug card 5755 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
- ChEMBL CHEMBL4594334 — compound record and bioactivity
- DrugBank DB16199 — drug record
- FDA UNII 0GRZ0F5XJ6 — substance identity
- DailyMed: FDA labels for Eplontersen — current prescribing information (label search)
- UniProt P02766 (TTR) — target protein
- PharmGKB / CPIC — pharmacogenomic label annotations
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