{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T13:47:08Z","timestamp":1790776028626,"version":"4.1.0"},"reference-count":20,"publisher":"Springer Science and Business Media LLC","issue":"16","license":[{"start":{"date-parts":[[2023,10,19]],"date-time":"2023-10-19T00:00:00Z","timestamp":1697673600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2023,10,19]],"date-time":"2023-10-19T00:00:00Z","timestamp":1697673600000},"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":[[2023,11]]},"DOI":"10.1007\/s40265-023-01955-9","type":"journal-article","created":{"date-parts":[[2023,10,19]],"date-time":"2023-10-19T04:02:16Z","timestamp":1697688136000},"page":"1551-1557","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["Pozelimab: First Approval"],"prefix":"10.1007","volume":"83","author":[{"given":"Sheridan M.","family":"Hoy","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2023,10,19]]},"reference":[{"issue":"2","key":"1955_CR1","doi-asserted-by":"publisher","first-page":"128","DOI":"10.1038\/s41590-020-00830-z","volume":"22","author":"A Ozen","year":"2021","unstructured":"Ozen A, Kasap N, Vujkovic-Cvijin I, et al. Broadly effective metabolic and immune recovery with C5 inhibition in CHAPLE disease. Nat Immunol. 2021;22(2):128\u201339.","journal-title":"Nat Immunol"},{"key":"1955_CR2","doi-asserted-by":"publisher","first-page":"17028","DOI":"10.1038\/nrdp.2017.28","volume":"3","author":"A Hill","year":"2017","unstructured":"Hill A, DeZern AE, Kinoshita T, et al. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017;3:17028.","journal-title":"Nat Rev Dis Primers"},{"issue":"1","key":"1955_CR3","doi-asserted-by":"publisher","first-page":"52","DOI":"10.1056\/NEJMoa1615887","volume":"377","author":"A Ozen","year":"2017","unstructured":"Ozen A, Comrie WA, Ardy RC, et al. CD55 deficiency, early-onset protein-losing enteropathy, and thrombosis. N Engl J Med. 2017;377(1):52\u201361.","journal-title":"N Engl J Med"},{"issue":"8","key":"1955_CR4","doi-asserted-by":"publisher","first-page":"733","DOI":"10.1056\/NEJMra2301594","volume":"389","author":"A Ozen","year":"2023","unstructured":"Ozen A, Lenardo MJ. Protein-losing enteropathy. N Engl J Med. 2023;389(8):733\u201348.","journal-title":"N Engl J Med"},{"key":"1955_CR5","unstructured":"Regeneron Pharmaceuticals Inc. VEOPOZ (pozelimab-bbfg) injection, for intravenous or subcutaneous use: US prescribing information. 2023. https:\/\/www.fda.gov\/. Accessed 23 Aug 2023."},{"key":"1955_CR6","unstructured":"Regeneron Pharmaceuticals Inc. Veopoz\u2122 (pozelimab-bbfg) receives FDA approval as the first treatment for children and adults with CHAPLE disease [media release]. 18 Aug 2023. https:\/\/www.regeneron.com\/."},{"key":"1955_CR7","unstructured":"US Food & Drug Administration. FDA approves first treatment for CD55-deficient protein-losing enteropathy (CHAPLE disease) [media release]. 18 Aug 2023. https:\/\/www.fda.gov\/drugs\/news-events-human-drugs\/fda-approves-first-treatment-cd55-deficient-protein-losing-enteropathy-chaple-disease."},{"issue":"5","key":"1955_CR8","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1371\/journal.pone.0231892","volume":"15","author":"A Latuszek","year":"2020","unstructured":"Latuszek A, Liu Y, Olsen O, et al. Inhibition of complement pathway activation with pozelimab, a fully human antibody to complement component C5. PLoS ONE. 2020;15(5):1\u201323.","journal-title":"PLoS ONE"},{"key":"1955_CR9","unstructured":"US Food & Drug Administration. Orphan drug designations and approvals. 2023. https:\/\/www.fda.gov\/. Accessed 13 Sep 2023."},{"key":"1955_CR10","unstructured":"Regeneron Pharmaceuticals Inc., Alnylam Pharmaceuticals Inc. Regeneron and Alnylam announce broad collaboration to discover, develop and commercialize RNAi therapeutics focused on ocular and central nervous system (CNS) diseases [media release]. 8 April 2019. http:\/\/www.regeneron.com."},{"key":"1955_CR11","unstructured":"United States Securities and Exchange Commission. Form 10-K: annual report pursuant to Section 13 OR 15(d) of the Securities Exchange Act of 1934 for the fiscal year ended December 31, 2022. Commission File Number 001-36407 (Alnylam Pharmaceuticals, Inc.). 2022. https:\/\/www.sec.gov\/Archives\/edgar\/data\/1178670\/000117867023000005\/alny-20221231.htm. Accessed 11 Sep 2023."},{"key":"1955_CR12","doi-asserted-by":"crossref","unstructured":"Weyne J, Ni Y, DelGizzi R, et al. A randomized, double-blind, placebo-controlled phase 1 study of the pharmacokinetics and pharmacodynamics of REGN3918, a human antibody against complement factor C5, in healthy volunteers [abstract no. 1039]. Blood. 2018;132(Suppl 1).","DOI":"10.1182\/blood-2018-99-112262"},{"key":"1955_CR13","doi-asserted-by":"crossref","unstructured":"Devalaraja-Narashimha K, Ni YG, Huang C, et al. Pozelimab, a human antibody against complement factor C5, demonstrates robust inhibition of alternative complement activity both in normal human serum and in phase I normal healthy volunteers [abstract no. 2278]. Blood. 2019;134(Suppl 1):2278.","DOI":"10.1182\/blood-2019-129865"},{"key":"1955_CR14","unstructured":"US Food & Drug Administration. Multi-disciplinary review. 2023. https:\/\/www.fda.gov\/. Accessed 27 Sep 2023."},{"key":"1955_CR15","doi-asserted-by":"crossref","unstructured":"Jang JH, Weyne J, Chaudhari U, et al. Pozelimab, a human monoclonal antibody against complement factor C5, provided inhibition of intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria [abstract no. 1128]. Blood. 2021;138(Suppl 1).","DOI":"10.1182\/blood-2021-146178"},{"key":"1955_CR16","doi-asserted-by":"crossref","unstructured":"Jang J, Wong R, Weyne J, et al. Long-term efficacy and safety of pozelimab monotherapy in patients with paroxysmal nocturnal hemoglobinuria [abstract no. P775]. HemaSphere. 2023;7(Supp; 3):1433\u201334.","DOI":"10.1097\/01.HS9.0000970004.43570.16"},{"key":"1955_CR17","unstructured":"US National Institutes of Health. ClinicalTrials.gov identifer NCT04162470. 2023. http:\/\/clinicaltrials.gov\/. Accessed 27 Sep 2023."},{"key":"1955_CR18","unstructured":"US National Institutes of Health. ClinicalTrials.gov identifier NCT04811716. 2023. http:\/\/clinicaltrials.gov\/. Accessed 27 Sep 2023."},{"key":"1955_CR19","doi-asserted-by":"crossref","unstructured":"Jang J, Wong R, Pavani R, et al. A phase 2, randomized trial evaluating the safety and efficacy of pozelimab and cemdisiran in patients with paroxysmal nocturnal hemoglobinuria [abstract no. P782]. HemaSphere. 2023;7(Suppl 3):1449\u201350.","DOI":"10.1097\/01.HS9.0000970032.01817.07"},{"key":"1955_CR20","doi-asserted-by":"crossref","unstructured":"Kelly R, Houghton N, Munir T, et al. A phase 2, open-label study evaluating the safety and efficacy of combination pozelimab and cemdisiran therapy in patients with paroxysmal nocturnal hemoglobinuria who switch from eculizumab [abstract no. P797]. HemaSphere. 2023;7(Suppl 3):1481\u201382.","DOI":"10.1097\/01.HS9.0000970092.68730.83"}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-023-01955-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40265-023-01955-9\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-023-01955-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,3]],"date-time":"2023-11-03T07:30:30Z","timestamp":1698996630000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40265-023-01955-9"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,10,19]]},"references-count":20,"journal-issue":{"issue":"16","published-print":{"date-parts":[[2023,11]]}},"alternative-id":["1955"],"URL":"https:\/\/doi.org\/10.1007\/s40265-023-01955-9","relation":{},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,10,19]]},"assertion":[{"value":"19 October 2023","order":1,"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. Sheridan M. Hoy 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"}}]}}