Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis.
[3] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca.
[4][5][6][7] Eplontersen was approved for medical use in the United States in December 2023 and in the UK in October 2024.
[3][8][9][10][11] Eplontersen is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
[3] The most common adverse reactions include decreased vitamin A and vomiting.