Eplontersen Explained

Width:300
Tradename:Wainua
Dailymedid:Eplontersen
Routes Of Administration:Subcutaneous
Atc Prefix:N07
Atc Suffix:XX21
Legal Ca:Rx-only
Legal Ca Comment:[1] [2]
Legal Us:Rx-only
Legal Us Comment:[3]
Cas Number:1637600-16-8
Drugbank:DB16199
Unii:0GRZ0F5XJ6
Kegg:D12754
Synonyms:AKCEA-TTR-LRx

Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. It is a transthyretin-directed antisense oligonucleotide. 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.[8] [9] [10]

Medical uses

Eplontersen is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Society and culture

Names

Eplontersen is the international nonproprietary name.[11]

Notes and References

  1. Web site: Register Of Innovative Drugs [Updated: 2024-07-04] ]. . 4 July 2024 . 15 July 2024.
  2. Web site: Notice: Multiple additions to the Prescription Drug List (PDL) [2024-08-13] ]. . 13 August 2024 . 15 August 2024.
  3. Web site: Wainua- eplontersen injection, solution . DailyMed . 21 December 2023 . 26 February 2024 . 26 February 2024 . https://web.archive.org/web/20240226055326/https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096 . live .
  4. Ionis announces FDA acceptance of New Drug Application for eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) . Ionis Pharmaceuticals . PR Newswire . 7 March 2023 . 21 December 2023 . 26 September 2023 . https://web.archive.org/web/20230926122608/https://www.prnewswire.com/news-releases/ionis-announces-fda-acceptance-of-new-drug-application-for-eplontersen-for-the-treatment-of-hereditary-transthyretin-mediated-amyloid-polyneuropathy-attrv-pn-301764027.html . live .
  5. Coelho . Teresa . Waddington Cruz . Márcia . Chao . Chi-Chao . Parman . Yeşim . Wixner . Jonas . Weiler . Markus . Barroso . Fabio A. . Dasgupta . Noel R. . Jung . Shiangtung W. . Schneider . Eugene . Viney . Nicholas J. . Dyck . P. James B. . Ando . Yukio . Gillmore . Julian D. . Khella . Sami . Gertz . Morie A. . Obici . Laura . Berk . John L. . Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen . Neurology and Therapy . February 2023 . 12 . 1 . 267–287 . 10.1007/s40120-022-00414-z. 36525140 . 9837340 . free .
  6. Coelho . Teresa . Marques . Wilson . Dasgupta . Noel R. . Chao . Chi-Chao . Parman . Yeşim . França . Marcondes Cavalcante . Guo . Yuh-Cherng . Wixner . Jonas . Ro . Long-Sun . Calandra . Cristian R. . Kowacs . Pedro A. . Berk . John L. . Obici . Laura . Barroso . Fabio A. . Weiler . Markus . Conceição . Isabel . Jung . Shiangtung W. . Buchele . Gustavo . Brambatti . Michela . Chen . Jersey . Hughes . Steven G. . Schneider . Eugene . Viney . Nicholas J. . Masri . Ahmad . Gertz . Morie R. . Ando . Yukio . Gillmore . Julian D. . Khella . Sami . Dyck . P. James B. . Waddington Cruz . Márcia . Mazzeo . Anna . Papagianni . Aikaterini . Dimachkie . Mazen . Zaganas . Ioannis . Gane . Edward . Luigetti . Marco . Galan Davila . Lucia . Mezei . Michelle . Gonzalez Moreno . Juan . Cintas . Pascal . Pareyson . Davide . Traub . Rebecca . Khoury . Julie . Estol . Conrado . Needham . Merrilee . Adams . David . Polydefkis . Michael . Brannagan . Thomas . Bril . Vera . Attarian . Shahram . Rugiero . Marcelo . Distad . Barbara . Zamba Papanicolaou . Eleni . Lin . Kon-Ping . Benson . Merrill . Scheinberg . Morton . Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy . The Journal of the American Medical Association . October 2023 . 330 . 15 . 1448–1458 . 10.1001/jama.2023.18688. 37768671 . 10540057 .
  7. Diep . John K. . Yu . Rosie Z. . Viney . Nicholas J. . Schneider . Eugene . Guo . Shuling . Henry . Scott . Monia . Brett . Geary . Richard . Wang . Yanfeng . Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis . British Journal of Clinical Pharmacology . December 2022 . 88 . 12 . 5389–5398 . 10.1111/bcp.15468. 35869634 . 250989659 .
  8. Web site: Eplontersen: FDA-Approved Drugs . U.S. Food and Drug Administration (FDA) . 21 December 2023.
  9. Wainua (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis . Ionis Pharmaceuticals, Inc. . 21 December 2023 . 22 December 2023 . 22 December 2023 . https://web.archive.org/web/20231222063825/https://ir.ionispharma.com/news-releases/news-release-details/wainuatm-eplontersen-granted-regulatory-approval-us-treatment . live .
  10. Wainua (eplontersen) granted first-ever regulatory approval in the US for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis . AstraZeneca US . 22 December 2023 . 22 December 2023 . 22 December 2023 . https://web.archive.org/web/20231222063922/https://www.astrazeneca-us.com/media/press-releases/2023/wainua-eplontersen-granted-first-ever-regulatory-approval-in-the-us-for-the-treatment-of-adults-with-polyneuropathy-of-hereditary-transthyretin-mediated-amyloidosis.html . live .
  11. ((World Health Organization)) . 2021 . International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85 . WHO Drug Information . 35 . 1 . 10665/340684 . free . World Health Organization .

This article is licensed under the GNU Free Documentation License. It uses material from the Wikipedia article "Eplontersen".

Except where otherwise indicated, Everything.Explained.Today is © Copyright 2009-2024, A B Cryer, All Rights Reserved. Cookie policy.