Eplontersen
Eplontersen is a GalNAc-conjugated antisense oligonucleotide designed to treat transthyretin (TTR) amyloidosis. It works by binding to and causing degradation of TTR mRNA, thereby reducing the production of TTR protein that forms harmful amyloid deposits in tissues. This medication is specifically indicated for patients with hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy.
Eplontersen reduces harmful TTR protein production to treat hereditary transthyretin-mediated amyloidosis with polyneuropathy.
Key facts at a glance
- ATC
- N07XX21
- RxNorm
- 2671939
- Generic
- Eplontersen
- Class
- atc-n07
- Brands
- Wainua
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- prefilled syringe
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
10 groupspregnancy
first trimesteravoid if possible
discontinue use during pregnancy unless benefits significantly outweigh risks
regular fetal monitoring including ultrasound assessments
Use with caution and monitor for adverse effects.
pregnancy
second trimesteravoid if possible
continue only if clearly needed with careful risk-benefit assessment
routine prenatal monitoring
Use with caution and monitor for adverse effects.
pregnancy
third trimesteravoid if possible
consider discontinuation near term
fetal monitoring and assessment of maternal conditions
Use with caution and monitor for adverse effects.
lactation
breastfeedingdiscontinue breastfeeding or discontinue drug
due to unknown excretion in breast milk and potential serious adverse reactions in nursing infants, a decision should be made whether to discontinue breastfeeding or discontinue therapy
monitor infant for adverse reactions
pediatric_age
children adolescentsnot recommended
not approved for use in pediatric patients; safety and efficacy not established in patients under 18 years of age
n/a
geriatric
elderly 65 years and olderno specific adjustment required
no dose adjustment required based on age alone; monitor renal function in patients with age-related decline
baseline and periodic renal function monitoring
renal_impairment
mild renal impairmentno adjustment needed
no dose adjustment recommended for mild renal impairment
routine monitoring of renal function
renal_impairment
moderate to severe renal impairmentconsider dose reduction
use with caution; limited data available for patients with severe renal impairment; consider dose adjustment based on clinical response and tolerability
more frequent renal function monitoring
Use with caution and monitor for adverse effects.
hepatic_impairment
mild hepatic impairmentuse with caution
limited data in patients with mild hepatic impairment; monitor liver function tests
baseline and periodic liver function tests
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate to severe hepatic impairmentnot recommended unless benefits outweigh risks
not studied in patients with moderate to severe hepatic impairment; use only if benefits justify risks
regular hepatic function monitoring
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Eplontersen is a transthyretin (TTR) directed antisense oligonucleotide inhibitor
Indication
FDA approved indication for Eplontersen
Mechanism of action
Reduces serum TTR protein levels to prevent amyloid deposition
FDA approval year
FDA approval date for Wainua (eplontersen)
Eplontersen is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in patients with polyneuropathy. This rare genetic condition causes abnormal TTR protein to form amyloid deposits that damage nerves and other organs.
Eplontersen is an antisense oligonucleotide conjugated to GalNAc (N-acetylgalactosamine), which targets liver cells where TTR protein is primarily produced. It binds to TTR messenger RNA (mRNA) and triggers its degradation, resulting in significantly reduced levels of both mutant and wild-type TTR protein in the blood and tissues.
As an antisense therapy, Eplontersen addresses the underlying cause of TTR amyloidosis by reducing protein production rather than just managing symptoms. It is administered via subcutaneous injection. Patients should be monitored for potential liver effects and adequate vitamin A intake is recommended, as TTR normally carries vitamin A in the blood.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy