World Clinical Drugs, Volume. 46, Issue 7, 715(2025)

A new drug for the treatment of hereditary transthyretin amyloid polyneuropathy: eplontersen

ZHU Haibin1, WANG Yue2, LIAO Xiaolan1, WU Tingting1, and YU Zicheng1
Author Affiliations
  • 1Department of Pharmacy, Yangpu Hospital, School of Medicine Tongji University, Shanghai 200090, China
  • 2Department of Pharmacy, Beijing Hospital/National Center of Gerontology, Institute of Geriatric Medicine/Chinese Academy of Medical Sciences, Beijing 100730, China
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    References(22)

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    [18] [18] DIEP JK, YU RZ, VINEY NJ,et al. Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis [J]. Br J Clin Pharmacol, 2022, 88(12): 5389-5398.

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    [21] [21] COELHO T, WADDINGTON CRUZ M, Chao CC,et al. Characteristics of patients with hereditary transthyretin amyloidosis-polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an open-label phase 3 study of eplontersen [J]. Neurol Ther, 2023, 12(1): 267-287.

    [22] [22] ClinicalTrials. A study to assess the long-term safety and efficacy of eplontersen (formerly known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in patients with hereditary transthyretin-mediated amyloid polyneuropathy [EB/OL]. (2023-10-06) [2024-12-16]. https://www.clinicaltrials.gov/study/NCT05071300.

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    [24] [24] ClinicalTrials. EPIC-ATTR: a study to evaluate the effect of eplontersen on the transthyretin reduction and long-term safety in Chinese subjects with transthyretin amyloid cardiomyopathy (EPIC-ATTR) [EB/OL]. (2024-01-08) [2024-12-16]. https://www.clinicaltrials.gov/study/NCT06194825.

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    ZHU Haibin, WANG Yue, LIAO Xiaolan, WU Tingting, YU Zicheng. A new drug for the treatment of hereditary transthyretin amyloid polyneuropathy: eplontersen[J]. World Clinical Drugs, 2025, 46(7): 715

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    Paper Information

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    Received: Dec. 28, 2024

    Accepted: Aug. 26, 2025

    Published Online: Aug. 26, 2025

    The Author Email:

    DOI:10.13683/j.wph.2025.07.015

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