{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,17]],"date-time":"2026-10-17T19:22:13Z","timestamp":1792264933553,"version":"4.4.0"},"reference-count":24,"publisher":"Springer Science and Business Media LLC","issue":"9","license":[{"start":{"date-parts":[[2023,5,16]],"date-time":"2023-05-16T00:00:00Z","timestamp":1684195200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2023,5,16]],"date-time":"2023-05-16T00:00:00Z","timestamp":1684195200000},"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":[[2023,6]]},"DOI":"10.1007\/s40265-023-01883-8","type":"journal-article","created":{"date-parts":[[2023,5,16]],"date-time":"2023-05-16T05:03:16Z","timestamp":1684213396000},"page":"819-824","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":44,"title":["Trofinetide: First Approval"],"prefix":"10.1007","volume":"83","author":[{"given":"Susan J.","family":"Keam","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2023,5,16]]},"reference":[{"key":"1883_CR1","doi-asserted-by":"publisher","first-page":"2813","DOI":"10.2147\/NDT.S371483","volume":"18","author":"BE Collins","year":"2022","unstructured":"Collins BE, Neul JL. Rett syndrome and MECP2 duplication syndrome: disorders of MeCP2 dosage. Neuropsychiatr Dis Treat. 2022;18:2813\u201335.","journal-title":"Neuropsychiatr Dis Treat"},{"issue":"1","key":"1883_CR2","doi-asserted-by":"publisher","DOI":"10.1136\/bmjpo-2020-000717","volume":"4","author":"C Fu","year":"2020","unstructured":"Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatr Open. 2020;4(1): e000717.","journal-title":"BMJ Paediatr Open."},{"issue":"2","key":"1883_CR3","doi-asserted-by":"publisher","DOI":"10.1098\/rsob.170216","volume":"8","author":"SM Kyle","year":"2018","unstructured":"Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018;8(2): 170216.","journal-title":"Open Biol"},{"key":"1883_CR4","doi-asserted-by":"publisher","DOI":"10.1016\/j.cct.2022.106704","volume":"114","author":"JL Neul","year":"2022","unstructured":"Neul JL, Percy AK, Benke TA, et al. Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome. Contemp Clin Trials. 2022. https:\/\/doi.org\/10.1016\/j.cct.2022.106704.","journal-title":"Contemp Clin Trials"},{"key":"1883_CR5","unstructured":"Neuren Pharmaceuticals Ltd. Annual Report 2021; 2022. https:\/\/www.neurenpharma.com\/. Accessed 28 Mar 2023."},{"issue":"2","key":"1883_CR6","doi-asserted-by":"publisher","first-page":"162","DOI":"10.1111\/dmcn.15383","volume":"65","author":"N Panayotis","year":"2023","unstructured":"Panayotis N, Ehinger Y, Felix MS, et al. State-of-the-art therapies for Rett syndrome. Dev Med Child Neurol. 2023;65(2):162\u201370.","journal-title":"Dev Med Child Neurol"},{"issue":"4","key":"1883_CR7","doi-asserted-by":"publisher","first-page":"554","DOI":"10.1021\/acschemneuro.2c00675","volume":"14","author":"SC Silva-Reis","year":"2023","unstructured":"Silva-Reis SC, Sampaio-Dias IE, Costa VM, et al. Concise overview of glypromate neuropeptide research: from chemistry to pharmacological applications in neurosciences. ACS Chem Neurosci. 2023;14(4):554\u201372.","journal-title":"ACS Chem Neurosci"},{"key":"1883_CR8","unstructured":"US FDA. FDA approves first treatment for Rett syndrome [media release]; 13 Mar 2023. https:\/\/www.fda.gov\/."},{"key":"1883_CR9","unstructured":"Acadia Pharmaceuticals Inc. Trofinetide: US prescribing information; 2023. https:\/\/daybue.com\/. Accessed 14 Mar 2023."},{"key":"1883_CR10","unstructured":"Neuren Pharmaceuticals Ltd. FDA approval of DAYBUE\u2122 (trofinetide)\u2013the first approved treatment for Rett syndrome [media release]; 13 Mar 2023. https:\/\/www.neurenpharma.com\/."},{"key":"1883_CR11","unstructured":"Neuren Pharmaceuticals Ltd. Neuren pipeline; 2023. https:\/\/www.neurenpharma.com\/. Accessed 29 Mar 2023."},{"key":"1883_CR12","doi-asserted-by":"publisher","first-page":"37","DOI":"10.1016\/j.pediatrneurol.2017.07.002","volume":"76","author":"DG Glaze","year":"2017","unstructured":"Glaze DG, Neul JL, Percy A, et al. A double-blind, randomized, placebo-controlled clinical study of trofinetide in the treatment of Rett syndrome. Pediatr Neurol. 2017;76:37\u201346.","journal-title":"Pediatr Neurol"},{"issue":"16","key":"1883_CR13","doi-asserted-by":"publisher","first-page":"e1912","DOI":"10.1212\/WNL.0000000000007316","volume":"92","author":"DG Glaze","year":"2019","unstructured":"Glaze DG, Neul JL, Kaufmann WE, et al. Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome. Neurology. 2019;92(16):e1912\u201325.","journal-title":"Neurology"},{"key":"1883_CR14","doi-asserted-by":"publisher","first-page":"30","DOI":"10.1016\/j.pediatrneurol.2020.04.019","volume":"110","author":"E Berry-Kravis","year":"2020","unstructured":"Berry-Kravis E, Horrigan JP, Tartaglia N, et al. A double-blind, randomized, placebo-controlled clinical study of trofinetide in the treatment of Fragile X syndrome. Pediatr Neurol. 2020;110:30\u201341.","journal-title":"Pediatr Neurol"},{"key":"1883_CR15","unstructured":"Acadia Pharmaceuticals Inc, Neuren Pharmaceuticals Ltd. Acadia Pharmaceuticals and Neuren Pharmaceuticals announce exclusive license agreement for the North American development and commercialization of trofinetide in Rett Syndrome [media release]; 6 Aug 2018. https:\/\/acadia.com\/."},{"key":"1883_CR16","unstructured":"Acadia Pharmaceuticals Inc. Form 10-K; 2022. https:\/\/ir.acadia.com\/. Accessed 28 Mar 2023."},{"issue":"1\u20132","key":"1883_CR17","doi-asserted-by":"publisher","first-page":"85","DOI":"10.1016\/j.jns.2008.12.003","volume":"278","author":"MJ Bickerdike","year":"2009","unstructured":"Bickerdike MJ, Thomas GB, Batchelor DC, et al. NNZ-2566: a Gly-Pro-Glu analogue with neuroprotective efficacy in a rat model of acute focal stroke. J Neurol Sci. 2009;278(1\u20132):85\u201390.","journal-title":"J Neurol Sci"},{"issue":"6","key":"1883_CR18","doi-asserted-by":"publisher","first-page":"2029","DOI":"10.1073\/pnas.0812394106","volume":"106","author":"D Tropea","year":"2009","unstructured":"Tropea D, Giacometti E, Wilson NR, et al. Partial reversal of Rett syndrome-like symptoms in MeCP2 mutant mice. Proc Natl Acad Sci USA. 2009;106(6):2029\u201334.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"1","key":"1883_CR19","doi-asserted-by":"publisher","first-page":"71","DOI":"10.1007\/s12017-015-8341-2","volume":"17","author":"RM Deacon","year":"2015","unstructured":"Deacon RM, Glass L, Snape M, et al. NNZ-2566, a novel analog of (1\u20133) IGF-1, as a potential therapeutic agent for Fragile X syndrome. Neuromolecular Med. 2015;17(1):71\u201382.","journal-title":"Neuromolecular Med"},{"issue":"Suppl 28","key":"1883_CR20","first-page":"S174","volume":"92","author":"M Darwish","year":"2022","unstructured":"Darwish M, Harlick J, Youakim J, et al. A phase 1, ascending dose study to assess the potential effects of trofinetide on QTc interval, safety and tolerability, and pharmacokinetics in healthy adults [abstract no. 212]. Ann Neurol. 2022;92(Suppl 28):S174.","journal-title":"Ann Neurol"},{"issue":"6","key":"1883_CR21","doi-asserted-by":"publisher","first-page":"513","DOI":"10.1007\/s40261-022-01156-4","volume":"42","author":"M Darwish","year":"2022","unstructured":"Darwish M, Youakim JM, Harlick J, et al. A phase 1, open-label study to evaluate the effects of food and evening dosing on the pharmacokinetics of oral trofinetide in healthy adult subjects. Clin Drug Investig. 2022;42(6):513\u201324.","journal-title":"Clin Drug Investig"},{"key":"1883_CR22","doi-asserted-by":"crossref","unstructured":"Percy A, Berry-Kravis E, Lieberman D, et al. Trofinetide for the treatment of Rett syndrome: an open-label study in girls 2 to 4 years of age [abstract no. P13.9-005]. In: American Academy of Neurology (AAN) Annual Meeting; 2023.","DOI":"10.1212\/WNL.0000000000201897"},{"issue":"Suppl 28","key":"1883_CR23","first-page":"S162","volume":"92","author":"J Neul","year":"2022","unstructured":"Neul J, Percy A, Benke T, et al. Efficacy and safety of trofinetide for the treatment of females with Rett syndrome: results from the randomized, double-blind, phase 3 LAVENDER study [abstract no. 191]. Ann Neurol. 2022;92(Suppl 28):S162\u20133.","journal-title":"Ann Neurol"},{"key":"1883_CR24","unstructured":"Acadia Pharmaceuticals Inc. 2023 (Data on file)."}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-023-01883-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40265-023-01883-8\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-023-01883-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,6,7]],"date-time":"2023-06-07T06:41:20Z","timestamp":1686120080000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40265-023-01883-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,5,16]]},"references-count":24,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2023,6]]}},"alternative-id":["1883"],"URL":"https:\/\/doi.org\/10.1007\/s40265-023-01883-8","relation":{},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,5,16]]},"assertion":[{"value":"16 May 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. Susan J. Keam is a contracted employee of Adis International Ltd\/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article 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"}}]}}