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# Ionis/AstraZeneca Planning NDA for Eplontersen
- URL: https://www.fdaweb.com/ionis-astrazeneca-planning-nda-for-eplontersen/
- Published: 2022-09-07T12:00:00.000Z
- Updated: 2026-09-14T17:58:34.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5152684

Ionis Pharmaceuticals and AstraZeneca plan to submit an NDA for eplontersen later this year for treating patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) based on new study results announced 9/7\. ATTRv is a severe, progressive, and life-threatening disease caused by the abnormal formation of the transthyretin (TTR) protein.

An interim analysis of the Phase 3 NEURO-TTRansform study of eplontersen found the therapy demonstrated a statistically significant and clinically meaningful change from baseline for its co-primary and secondary efficacy endpoints compared to a placebo, according to the companies. Eplontersen also demonstrated a favorable safety and tolerability profile, they say.

In the study, eplontersen achieved an 81.2% mean reduction in the co-primary endpoint of serum TRR concentration compared to baseline, demonstrating reduced TTR protein production, Ionis and AstraZeneca say. The therapy also demonstrated a significant treatment effect on the co-primary endpoint of modified Neuropathy Impairment Score +7 with a statistically significant difference in mean change from baseline for those treated with eplontersen versus placebo.

Eplontersen has been [granted an orphan drug designation](https://c212.net/c/link/?t=0&l=en&o=3640119-1&h=2462827524&u=https%3A%2F%2Fir.ionispharma.com%2Fnews-releases%2Fnews-release-details%2Fionis-announces-eplontersen-receives-orphan-drug-designation-us&a=granted+Orphan+Drug+Designation&ref=fdaweb.com). It is also currently being evaluated in the Phase 3 CARDIO-TTRansform study for amyloid transthyretin cardiomyopathy, a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.