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Brand Name :
N/A
Synonyms :
eplontersen
Class :
Rheumatologics, Antisense Oligonucleotides
Hereditary Transthyretin Amyloidosis
FDA Approval Pending for hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN)
Hereditary Transthyretin Amyloidosis
Administer dose of 45 mg subcutaneously one time in a month
Not suggested
Refer to adult dosing
Actions and Spectrum
eplontersen is designed to bind to the RNA sequence of the TTR gene, forming a complementary strand.
None
Black Box Warning:
None
Contraindication/Caution:
None
Pregnancy warnings:
Pregnancy category: N/A
Lactation: Excretion into human milk is unknown
Pregnancy Categories:
Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester.
Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women.
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.
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.
Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.
Category N: There is no data available for the drug under this category.
Pharmacology
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.
Pharmacodynamics
Limited information available
Pharmacokinetics
Limited information available on ADME
Administration
eplontersen is administered through subcutaneous injection.
Patient information leaflet
Generic Name: eplontersen (FDA Approval Pending)
Why do we use eplontersen?
eplontersen is used for the treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy.