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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.[1] [6] [7] [8]
Medical uses [ edit ]
Eplontersen is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.[1]
Society and culture [ edit ]
Eplontersen is the international nonproprietary name .[9]
References [ edit ]
^ 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 .
^ 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 .
^ 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 .
^ "Eplontersen: FDA-Approved Drugs" . U.S. Food and Drug Administration (FDA) . Retrieved 21 December 2023 .
^ "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 .
^ "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 .
^ World Health Organization (2021). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information . 35 (1 ). hdl :10665/340684 .
External links [ edit ]
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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R e t r i e v e d f r o m " https://en.wikipedia.org/w/index.php?title=Eplontersen&oldid=1230793538 "
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