{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T15:59:45Z","timestamp":1790870385924,"version":"4.1.0"},"reference-count":16,"publisher":"Springer Science and Business Media LLC","issue":"4","license":[{"start":{"date-parts":[[2024,2,28]],"date-time":"2024-02-28T00:00:00Z","timestamp":1709078400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2024,2,28]],"date-time":"2024-02-28T00:00:00Z","timestamp":1709078400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Drugs"],"published-print":{"date-parts":[[2024,4]]},"DOI":"10.1007\/s40265-024-02008-5","type":"journal-article","created":{"date-parts":[[2024,2,28]],"date-time":"2024-02-28T03:02:27Z","timestamp":1709089347000},"page":"473-478","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":53,"title":["Eplontersen: First Approval"],"prefix":"10.1007","volume":"84","author":[{"given":"Tina","family":"Nie","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,2,28]]},"reference":[{"key":"2008_CR1","unstructured":"Ionis Pharmaceuticals Inc. WAINUA\u2122 (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis [media release]. 21 Dec 2023. http:\/\/www.ionispharma.com\/."},{"issue":"2","key":"2008_CR2","doi-asserted-by":"publisher","first-page":"127","DOI":"10.1007\/s40259-023-00577-7","volume":"37","author":"A Ioannou","year":"2023","unstructured":"Ioannou A, Fontana M, Gillmore JD. RNA targeting and gene editing strategies for transthyretin amyloidosis. BioDrugs. 2023;37(2):127\u201342.","journal-title":"BioDrugs"},{"issue":"1","key":"2008_CR3","doi-asserted-by":"publisher","first-page":"652","DOI":"10.1002\/ehf2.13154","volume":"8","author":"NJ Viney","year":"2021","unstructured":"Viney NJ, Guo S, Tai LJ, et al. Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data. ESC Heart Fail. 2021;8(1):652\u201361.","journal-title":"ESC Heart Fail."},{"issue":"13","key":"2008_CR4","doi-asserted-by":"publisher","first-page":"1371","DOI":"10.1007\/s40265-018-0968-5","volume":"78","author":"SJ Keam","year":"2018","unstructured":"Keam SJ. Inotersen: first global approval. Drugs. 2018;78(13):1371\u20136.","journal-title":"Drugs"},{"issue":"15","key":"2008_CR5","doi-asserted-by":"publisher","first-page":"1448","DOI":"10.1001\/jama.2023.18688","volume":"330","author":"T Coelho","year":"2023","unstructured":"Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy. JAMA. 2023;330(15):1448\u201358.","journal-title":"JAMA"},{"key":"2008_CR6","unstructured":"AstraZeneca Pharmaceuticals LP. WAINUA\u2122 (eplontersen) injection, for subcutaneous use. 2023. https:\/\/www.accessdata.fda.gov\/. Accessed 09 Feb 2024."},{"key":"2008_CR7","unstructured":"Ionis Pharmaceuticals Inc. Ionis and AstraZeneca to develop and commercialize eplontersen [media release]. 06 Dec 2021. http:\/\/www.ionispharma.com\/."},{"key":"2008_CR8","unstructured":"Ionis Pharmaceuticals Inc. Ionis expands eplontersen agreement with AstraZeneca to include exclusive rights in Latin America [media release]. 28 Jul 2023. http:\/\/www.ionispharma.com\/."},{"key":"2008_CR9","unstructured":"Ionis Pharmaceuticals Inc. Ionis and AstraZeneca close deal to develop and commercialize eplontersen [media release]. 28 Dec 2021. http:\/\/www.ionispharma.com\/."},{"key":"2008_CR10","unstructured":"Ionis Pharmaceuticals Inc. Ionis and Akcea partner to commercialize inotersen for hATTR [media release]. 15 Mar 2018. http:\/\/www.ionispharma.com\/."},{"key":"2008_CR11","unstructured":"Ionis Pharmaceuticals Inc. Ionis Pharmaceuticals completes acquisition of Akcea Therapeutics [media release]. 12 Oct 2020. http:\/\/www.ionispharma.com\/."},{"key":"2008_CR12","unstructured":"Ionis Pharmaceuticals Inc. Form 10-K to the United States Securities and Exchange Commission. 2021. https:\/\/www.sec.gov\/. Accessed 09 Feb 2024."},{"issue":"12","key":"2008_CR13","doi-asserted-by":"publisher","first-page":"5389","DOI":"10.1111\/bcp.15468","volume":"88","author":"JK Diep","year":"2022","unstructured":"Diep JK, Yu RZ, Viney NJ, et al. Population pharmacokinetic\/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis. Br J Clin Pharmacol. 2022;88(12):5389\u201398.","journal-title":"Br J Clin Pharmacol"},{"issue":"1","key":"2008_CR14","doi-asserted-by":"publisher","first-page":"22","DOI":"10.1056\/NEJMoa1716793","volume":"379","author":"MD Benson","year":"2018","unstructured":"Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):22\u201331.","journal-title":"N Engl J Med"},{"issue":"Suppl 1","key":"2008_CR15","doi-asserted-by":"publisher","first-page":"5764","DOI":"10.1182\/blood-2019-129269","volume":"134","author":"RH Falk","year":"2019","unstructured":"Falk RH, Gertz MA, Benson MD, et al. Rationale and design of a phase 3 study to evaluate the efficacy and safety of ION-682884 in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) [abstract]. Blood. 2019;134(Suppl 1):5764.","journal-title":"Blood"},{"key":"2008_CR16","unstructured":"US National Institutes of Health. ClinicalTrials.gov identifier NCT04136171. 2024. https:\/\/www.clinicaltrials.gov\/. Accessed 09 Feb 2024."}],"updated-by":[{"DOI":"10.1007\/s40265-024-02024-5","type":"correction","label":"Correction","source":"publisher","updated":{"date-parts":[[2024,3,28]],"date-time":"2024-03-28T00:00:00Z","timestamp":1711584000000}}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-024-02008-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40265-024-02008-5\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-024-02008-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,5,18]],"date-time":"2024-05-18T03:39:05Z","timestamp":1716003545000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40265-024-02008-5"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,2,28]]},"references-count":16,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024,4]]}},"alternative-id":["2008"],"URL":"https:\/\/doi.org\/10.1007\/s40265-024-02008-5","relation":{"correction":[{"id-type":"doi","id":"10.1007\/s40265-024-02024-5","asserted-by":"object"}]},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,2,28]]},"assertion":[{"value":"28 February 2024","order":1,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"28 March 2024","order":2,"name":"change_date","label":"Change Date","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Correction","order":3,"name":"change_type","label":"Change Type","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"A Correction to this paper has been published:","order":4,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"https:\/\/doi.org\/10.1007\/s40265-024-02024-5","URL":"https:\/\/doi.org\/10.1007\/s40265-024-02024-5","order":5,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The preparation of this review was not supported by any external funding.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Funding"}},{"value":"During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Tina Nie is a salaried employee of Adis International Ltd\/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Authorship and Conflict of interest"}},{"value":"Not applicable.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability"}}]}}