Key Information
What is eplontersen?
Eplontersen is used for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN or ATTRv-PN), which is a rare, progressive disease that can be fatal if not treated. Amyloidosis is a disease caused by a buildup of abnormal proteins called amyloid. Eplontersen works by decreasing the amount of TTR protein that is made, which slows disease progression and improves neuropathy and the patient's quality of life. Eplontersen brand name is Wainua.external link, opens in a new tab
Eplontersen drug class of medications called LICA (ligand-conjugated antisense oligonucleotide). Eplontersen mechanism of action is by causing the breakdown of mutant and wild-type TTR mRNA by binding to the TTR mRNA, which lowers serum TTR protein and TTR protein deposits in tissues.