Chemistry:Eplontersen

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Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis.[1] It is a transthyretin-directed antisense oligonucleotide.[1] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca.[2][3][4][5]

Eplontersen was approved for medical use in the United States in December 2023 and in the UK in October 2024.[1][6][7][8][9]

Medical uses

Eplontersen is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.[1]

Side effects

The most common adverse reactions include decreased vitamin A and vomiting.[1]

Society and culture

In October 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Wainzua, intended for the treatment of adults with hereditary transthyretin-mediated amyloidosis (ATTRv) and stage 1 or 2 polyneuropathy.[10] The applicant for this medicinal product is AstraZeneca AB.[10][11] Eplontersen was authorized for medical use in the European Union in March 2025.[10][12]

Names

Eplontersen is the international nonproprietary name.[13]

References

  1. ↑ 1.0 1.1 1.2 1.3 1.4 Cite error: Invalid <ref> tag; no text was provided for refs named Wainua FDA label
  2. ↑ "Ionis announces FDA acceptance of New Drug Application for eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)" (Press release). Ionis Pharmaceuticals. 7 March 2023. Archived from the original on 26 September 2023. Retrieved 21 December 2023 – via PR Newswire.
  3. ↑ Coelho, Teresa; Waddington Cruz, Márcia; Chao, Chi-Chao; Parman, Yeşim; Wixner, Jonas; Weiler, Markus; Barroso, Fabio A.; Dasgupta, Noel R. et al. (February 2023). "Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen". Neurology and Therapy 12 (1): 267–287. doi:10.1007/s40120-022-00414-z. PMID 36525140. 
  4. ↑ Coelho, Teresa; Marques, Wilson; Dasgupta, Noel R.; Chao, Chi-Chao; Parman, Yeşim; França, Marcondes Cavalcante; Guo, Yuh-Cherng; Wixner, Jonas et al. (October 2023). "Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy". The Journal of the American Medical Association 330 (15): 1448–1458. doi:10.1001/jama.2023.18688. PMID 37768671. 
  5. ↑ Diep, John K.; Yu, Rosie Z.; Viney, Nicholas J.; Schneider, Eugene; Guo, Shuling; Henry, Scott; Monia, Brett; Geary, Richard et al. (December 2022). "Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis". British Journal of Clinical Pharmacology 88 (12): 5389–5398. doi:10.1111/bcp.15468. PMID 35869634. 
  6. ↑ "Eplontersen: FDA-Approved Drugs". https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=217388. 
  7. ↑ "Wainua (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis". Ionis Pharmaceuticals, Inc. (Press release). 21 December 2023. Archived from the original on 22 December 2023. Retrieved 22 December 2023.
  8. ↑ "Wainua (eplontersen) granted first-ever regulatory approval in the US for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis". AstraZeneca US (Press release). 22 December 2023. Archived from the original on 22 December 2023. Retrieved 22 December 2023.
  9. ↑ "Eplontersen approved to treat adults with rare inherited nerve disease or polyneuropathy" (in en). 2024-10-16. https://www.gov.uk/government/news/eplontersen-approved-to-treat-adults-with-rare-inherited-nerve-disease-or-polyneuropathy. 
  10. ↑ 10.0 10.1 10.2 "Wainzua EPAR". 17 October 2024. https://www.ema.europa.eu/en/medicines/human/EPAR/wainzua.  Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  11. ↑ "Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 October 2024". 18 October 2024. https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-14-17-october-2024. 
  12. ↑ "Wainzua PI". 7 March 2025. https://ec.europa.eu/health/documents/community-register/html/h1875.htm. 
  13. ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information 35 (1). 2021. 
  • Clinical trial number NCT04136184 for "NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy" at ClinicalTrials.gov
  • Clinical trial number NCT01737398 for "Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy" at ClinicalTrials.gov



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