
WAINUA (eplontersen) is a transthyretin-directed antisense oligonucleotide approved for treating polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). By degrading TTR mRNA, it reduces the production of abnormal TTR protein, slowing disease progression.
Administered monthly via subcutaneous injection, WAINUA requires vitamin A supplementation due to its effect on serum vitamin A levels. Clinical trials demonstrated significant improvements in neuropathy and quality of life compared to placebo. Its safety profile includes manageable adverse effects such as decreased vitamin A and vomiting.