Eplontersen Sodium

Treats polyneuropathy of hereditary transthyretin-mediated amyloidosis. Sold as Wainua. Every FDA application for this active ingredient.

As of Nov 15, 2026, FDA lists 1 application for Eplontersen Sodium (sold as Wainua), first approved Dec 21, 2023, with 1 new use approved and 0 published complete response letters.

First approved: Dec 21, 2023
Applications: 1
New uses approved: 1
Complete response letters: 0
Medicare spending, 2025: $187M
Orphan exclusivity until: Dec 21, 2030
First approved in the EU: Mar 6, 2025

Medicare Spending

CMS Part D and Part B, 2025, before manufacturer rebates; matched by generic name

Product	Part	Spending, 2025	Change	Beneficiaries
Wainua	Part D	$187M	+228%	512

Who Was in the Trials

140 participants in the largest population FDA reports for the Dec 21, 2023 approval, from FDA's Drug Trials Snapshot; see every snapshot

Women: 31%
65 and older: 5%
White: 77%
Black: 4%
Asian: 16%
Hispanic or Latino: 16%

Orphan Designations (FDA)

One designation, 1 approved for the designated use; see all orphan designations

Treatment of transthyretin-mediated amyloidosisIonis Pharmaceuticals, Inc. · designated Jan 6, 2022 · approved Dec 21, 2023 · exclusive to Dec 21, 2030

Approvals in the EU (EMA)

First approved in the EU 14 months after the US. Centrally authorised medicines, matched by ingredient; see all EU approvals

Wainzua, new substanceAstraZeneca AB · Mar 6, 2025

Applications

NDAs, BLAs and generic ANDAs for this ingredient, oldest first; each opens its full history

Approved	Application	Sponsor	Supplements	Marketing
Dec 21, 2023	WainuaNDA 217388 · New molecular entity	Astrazeneca AB	4	Marketed

Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA