{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,27]],"date-time":"2026-10-27T16:20:55Z","timestamp":1793118055362,"version":"4.5.0"},"reference-count":11,"publisher":"Springer Science and Business Media LLC","issue":"8","license":[{"start":{"date-parts":[[2025,6,28]],"date-time":"2025-06-28T00:00:00Z","timestamp":1751068800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,6,28]],"date-time":"2025-06-28T00:00:00Z","timestamp":1751068800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Drugs"],"published-print":{"date-parts":[[2025,8]]},"DOI":"10.1007\/s40265-025-02203-y","type":"journal-article","created":{"date-parts":[[2025,6,27]],"date-time":"2025-06-27T22:24:24Z","timestamp":1751063064000},"page":"1073-1077","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":30,"title":["Fitusiran: First Approval"],"prefix":"10.1007","volume":"85","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6519-7831","authenticated-orcid":false,"given":"Arnold","family":"Lee","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,6,28]]},"reference":[{"key":"2203_CR1","doi-asserted-by":"publisher","first-page":"317","DOI":"10.2147\/JBM.S390521","volume":"14","author":"S Boyce","year":"2023","unstructured":"Boyce S, Rangarajan S. RNAi for the treatment of people with hemophilia: current evidence and patient selection. J Blood Med. 2023;14:317\u201327.","journal-title":"J Blood Med."},{"issue":"4","key":"2203_CR2","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.rpth.2023.100179","volume":"7","author":"G Young","year":"2023","unstructured":"Young G, Lenting PJ, Croteau SE, et al. Antithrombin-lowering in hemophilia: a closer look at fitusiran. Res Pract Thromb Haemost. 2023;7(4):1\u201313.","journal-title":"Res Pract Thromb Haemost."},{"key":"2203_CR3","unstructured":"Sanofi. Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors [media release]. 28 Mar 2025. https:\/\/www.globenewswire.com\/news-release\/2025\/03\/28\/3051637\/0\/en\/Press-Release-Qfitlia-approved-as-the-first-therapy-in-the-US-to-treat-hemophilia-A-or-B-with-or-without-inhibitors.html."},{"key":"2203_CR4","unstructured":"Genzyme Corporation. QFITLIA (fitusiran) injection, for subcutaneous use. 2025. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2025\/219019s000lbl.pdf. Accessed 1 Apr 2025."},{"key":"2203_CR5","unstructured":"Sanofi Genzyme. Sanofi Genzyme and Alnylam initiate ATLAS phase 3 program with investigational RNAi therapeutic fitusiran in patients with hemophilia A and B with or without Inhibitors [media release]. 7 Jul 2017. http:\/\/www.sanofigenzyme.com."},{"key":"2203_CR6","unstructured":"Sanofi. Sanofi and Alnylam conclude research and option phase of 2014 rare disease alliance [media release]. 8 Apr 2019. http:\/\/www.sanofi.com."},{"issue":"2","key":"2203_CR7","doi-asserted-by":"publisher","first-page":"227","DOI":"10.1182\/blood.2023023404","volume":"144","author":"S Verhenne","year":"2024","unstructured":"Verhenne S, McCluskey G, Maynadi\u00e9 H, et al. Fitusiran reduces bleeding in factor X-deficient mice. Blood. 2024;144(2):227\u201336.","journal-title":"Blood"},{"issue":"10386","key":"2203_CR8","doi-asserted-by":"publisher","first-page":"1427","DOI":"10.1016\/S0140-6736(23)00284-2","volume":"401","author":"G Young","year":"2023","unstructured":"Young G, Srivastava A, Kavakli K, et al. Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial. Lancet. 2023;401(10386):1427\u201337.","journal-title":"Lancet"},{"issue":"5","key":"2203_CR9","doi-asserted-by":"publisher","first-page":"e322","DOI":"10.1016\/S2352-3026(23)00037-6","volume":"10","author":"A Srivastava","year":"2023","unstructured":"Srivastava A, Rangarajan S, Kavakli K, et al. Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A\/B): a multicentre, open-label, randomised, phase 3 trial. Lancet Haematol. 2023;10(5):e322\u201332.","journal-title":"Lancet Haematol."},{"key":"2203_CR10","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2024027008","author":"G Young","year":"2025","unstructured":"Young G, Kavakli K, Klamroth R, et al. Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study. Blood. 2025. https:\/\/doi.org\/10.1182\/blood.2024027008.","journal-title":"Blood"},{"issue":"22","key":"2203_CR11","doi-asserted-by":"publisher","first-page":"2256","DOI":"10.1182\/blood.2023021864","volume":"143","author":"G Kenet","year":"2024","unstructured":"Kenet G, Nolan B, Zulfikar B, et al. Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA\/CFC prophylaxis: the ATLAS-PPX trial. Blood. 2024;143(22):2256\u201369.","journal-title":"Blood"}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-025-02203-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40265-025-02203-y\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-025-02203-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,9,6]],"date-time":"2025-09-06T23:58:33Z","timestamp":1757203113000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40265-025-02203-y"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,6,28]]},"references-count":11,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2025,8]]}},"alternative-id":["2203"],"URL":"https:\/\/doi.org\/10.1007\/s40265-025-02203-y","relation":{},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,6,28]]},"assertion":[{"value":"2 June 2025","order":1,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"28 June 2025","order":2,"name":"first_online","label":"First Online","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. Arnold Lee is a contracted 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"}}]}}