Eplontersen (Wainua™) is a ligand-conjugated antisense oligonucleotide developed by Ionis Pharmaceuticals and AstraZeneca for the treatment of TTR-mediated amyloidosis (ATTR). It targets *TTR* mRNA in the liver, reducing circulating TTR protein levels and amyloid deposition. In a phase III trial, subcutaneous eplontersen reduced serum TTR levels, inhibited neuropathy progression, and improved health-related quality of life in patients with hereditary ATTR polyneuropathy (ATTRv-PN). Based on these results, eplontersen received its first approval in the USA on December 21, 2023, for the treatment of ATTRv-PN. It is currently under regulatory review in the EU, UK, Switzerland, and Canada for the same indication. Additionally, eplontersen is in phase III development for ATTR cardiomyopathy.Eplontersen (Wainua™) is a ligand-conjugated antisense oligonucleotide developed by Ionis Pharmaceuticals and AstraZeneca for the treatment of TTR-mediated amyloidosis (ATTR). It targets *TTR* mRNA in the liver, reducing circulating TTR protein levels and amyloid deposition. In a phase III trial, subcutaneous eplontersen reduced serum TTR levels, inhibited neuropathy progression, and improved health-related quality of life in patients with hereditary ATTR polyneuropathy (ATTRv-PN). Based on these results, eplontersen received its first approval in the USA on December 21, 2023, for the treatment of ATTRv-PN. It is currently under regulatory review in the EU, UK, Switzerland, and Canada for the same indication. Additionally, eplontersen is in phase III development for ATTR cardiomyopathy.