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# FDA Approves Ionis/AstraZeneca’s Wainua
- URL: https://www.fdaweb.com/fda-approves-ionis-astrazenecas-wainua/
- Published: 2024-01-02T12:00:00.000Z
- Updated: 2026-09-14T14:19:21.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156015

FDA has approved Ionis Pharmaceuticals/AstraZeneca’s Wainua (eplontersen) to treat adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis. An Ionis [news release](https://www.prnewswire.com/news-releases/wainua-eplontersen-granted-regulatory-approval-in-the-us-for-the-treatment-of-adults-with-polyneuropathy-of-hereditary-transthyretin-mediated-amyloidosis-302021385.html?ref=fdaweb.com) says the approval was based on the Phase 3 NEURO-TTRansform trial that showed Wainua demonstrated consistent and sustained benefit halting neuropathy disease progression and improving neuropathy impairment and quality of life.

The release says Wainua is the only approved medicine to treat the condition that can be self-administered via an auto-injector.

Wainua was granted orphan drug designation by FDA to treat transthyretin-mediated amyloidosis. It is currently being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy, Ionis says.

Wainua is described as a ligand-conjugated antisense medicine designed to inhibit the production of transthyretin.