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November 22, 2025
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Brand Name :
N/A
Synonyms :
eplontersen
Class :
Rheumatologics, Antisense Oligonucleotides
Brand Name :
N/A
Synonyms :
eplontersen
Class :
Rheumatologics, Antisense Oligonucleotides
Hereditary Transthyretin AmyloidosisÂ
FDA Approval Pending for hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN)
Hereditary Transthyretin AmyloidosisÂ
Administer dose of 45 mg subcutaneously one time in a month
Not suggested Â
Refer to adult dosingÂ
Actions and Spectrum Â
eplontersen is designed to bind to the RNA sequence of the TTR gene, forming a complementary strand.
None
Black Box Warning:Â
NoneÂ
Contraindication/Caution:Â Â Â
None
Pregnancy warnings:    Â
Pregnancy category: N/AÂ
Lactation: Excretion into human milk is unknown Â
Pregnancy Categories:        Â
Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester.Â
Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women.  Â
Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.Â
Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.Â
Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.Â
Category N: There is no data available for the drug under this category.
Pharmacology Â
Antisense oligonucleotide that binds to the transthyretin (TTR) mRNA and degrades both mutant and wild-type TTR mRNA, reducing serum TTR protein and tissue TTR protein deposits.Â
PharmacodynamicsÂ
Limited information available Â
PharmacokineticsÂ
Limited information available on ADME
Administration Â
eplontersen is administered through subcutaneous injection.
Patient information leafletÂ
Generic Name: eplontersen (FDA Approval Pending)Â
Why do we use eplontersen?Â
eplontersen is used for the treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy.
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