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Eplontersen

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Eplontersen
Clinical data
Trade namesWainua
Other namesAKCEA-TTR-LRx
AHFS/Drugs.comEplontersen
License data
Routes of
administration		Subcutaneous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG

Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis.[2] It is a transthyretin-directed antisense oligonucleotide.[2] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca.[3][4][5][6]

Eplontersen was approved for medical use in the United States in December 2023 and in the UK in October 2024.[2][7][8][9][10]

Medical uses

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Eplontersen is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.[2]

Side effects

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The most common adverse reactions include decreased vitamin A and vomiting.[2]

Society and culture

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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.[11] The applicant for this medicinal product is AstraZeneca AB.[11][12]

Names

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Eplontersen is the international nonproprietary name.[13]

References

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  1. ^ "Summary Basis of Decision for Wainua". Drug and Health Products Portal. 1 September 2012. Retrieved 13 November 2024.
  2. ^ a b c d e f "Wainua- eplontersen injection, solution". DailyMed. 21 December 2023. Archived from the original on 26 February 2024. Retrieved 26 February 2024.
  3. ^ "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.
  4. ^ Coelho, Teresa; Waddington Cruz, Márcia; Chao, Chi-Chao; Parman, Yeşim; Wixner, Jonas; Weiler, Markus; 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. PMC 9837340. PMID 36525140.
  5. ^ Coelho, Teresa; Marques, Wilson; Dasgupta, Noel R.; Chao, Chi-Chao; Parman, Yeşim; França, Marcondes Cavalcante; 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. PMC 10540057. PMID 37768671.
  6. ^ Diep, John K.; Yu, Rosie Z.; Viney, Nicholas J.; Schneider, Eugene; Guo, Shuling; Henry, Scott; 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. S2CID 250989659.
  7. ^ "Eplontersen: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Retrieved 21 December 2023.
  8. ^ "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.
  9. ^ "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.
  10. ^ "Eplontersen approved to treat adults with rare inherited nerve disease or polyneuropathy". GOV.UK. 16 October 2024. Retrieved 21 October 2024.
  11. ^ a b "Wainzua EPAR". European Medicines Agency (EMA). 17 October 2024. Retrieved 19 October 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  12. ^ "Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 October 2024". European Medicines Agency (EMA). 18 October 2024. Retrieved 21 October 2024.
  13. ^ World Health Organization (2021). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information. 35 (1). hdl:10665/340684.
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  • 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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