Eplontersen Sodium

CHEMBL4594335 Phase 4 Approuvé Oligonucleotide
Half-Life
Bioavailability
Protein Binding
Molecular Weight
g/mol
LogP
Phase
4

This RNA-targeted therapy reduces the production of a harmful protein called transthyretin that builds up and damages the heart and nerves in people with a rare inherited condition. It is given as a subcutaneous injection.

Mécanisme d'action

Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mécanisme

Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

HBD / HBA

- / -

No targets recorded

Target interaction data is not yet available for this drug.

No interactions recorded

Drug interaction data is not yet available for this compound.

No side effects recorded

Side effect data is not yet available for this drug.

Foire aux questions

This RNA-targeted therapy reduces the production of a harmful protein called transthyretin that builds up and damages the heart and nerves in people with a rare inherited condition. It is given as a subcutaneous injection.

Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

Yes, Eplontersen Sodium is an approved drug. It has reached clinical phase 4. It is classified as a Oligonucleotide.

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References & Data Sources

  • ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4594335. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.

Avertissement médical

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.