{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T05:38:52Z","timestamp":1792820332303,"version":"4.5.0"},"reference-count":17,"publisher":"Springer Science and Business Media LLC","issue":"2","license":[{"start":{"date-parts":[[2021,1,6]],"date-time":"2021-01-06T00:00:00Z","timestamp":1609891200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2021,1,6]],"date-time":"2021-01-06T00:00:00Z","timestamp":1609891200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Drugs"],"published-print":{"date-parts":[[2021,2]]},"DOI":"10.1007\/s40265-020-01463-0","type":"journal-article","created":{"date-parts":[[2021,1,6]],"date-time":"2021-01-06T05:08:55Z","timestamp":1609909735000},"page":"277-282","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":287,"title":["Lumasiran: First Approval"],"prefix":"10.1007","volume":"81","author":[{"given":"Lesley J.","family":"Scott","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan J.","family":"Keam","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,1,6]]},"reference":[{"issue":"2","key":"1463_CR1","doi-asserted-by":"publisher","first-page":"494","DOI":"10.1681\/ASN.2016030338","volume":"28","author":"A Liebow","year":"2017","unstructured":"Liebow A, Li X, Racie T, et al. An investigational RNAi therapeutic targeting glycolate oxidase reduces oxalate production in models of primary hyperoxaluria. J Am Soc Nephrol. 2017;28(2):494\u2013503.","journal-title":"J Am Soc Nephrol."},{"issue":"7","key":"1463_CR2","doi-asserted-by":"publisher","first-page":"649","DOI":"10.1056\/NEJMra1301564","volume":"369","author":"P Cochat","year":"2013","unstructured":"Cochat P, Rumsby G. Primary hyperoxaluria. N Engl J Med. 2013;369(7):649\u201358.","journal-title":"N Engl J Med."},{"issue":"10","key":"1463_CR3","doi-asserted-by":"publisher","first-page":"673","DOI":"10.1038\/s41573-020-0075-7","volume":"19","author":"TC Roberts","year":"2020","unstructured":"Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nat Rev Drug Discov. 2020;19(10):673\u201394.","journal-title":"Nat Rev Drug Discov."},{"issue":"8","key":"1463_CR4","doi-asserted-by":"publisher","first-page":"1759","DOI":"10.1016\/j.ymthe.2020.06.015","volume":"28","author":"AJ Debacker","year":"2020","unstructured":"Debacker AJ, Voutila J, Catley M, et al. Delivery of oligonucleotides to the liver with GalNAc: from research to registered therapeutic drug. Mol Ther. 2020;28(8):1759\u201371.","journal-title":"Mol Ther."},{"key":"1463_CR5","unstructured":"European Commission. Commission implementing decision of 19.11.20 granting marketing authorisation under Regulation (EC) No 726\/2004 of the European Parliament and of the Council for \"Oxlumo\u2014lumasiran, an orphan medicinal product for human use. 2020. http:\/\/ec.europa.eu\/health\/documents\/community-register\/html\/h1496.htm. Accessed 24 Nov 2020."},{"key":"1463_CR6","unstructured":"US FDA. FDA approves first drug to treat rare metabolic disorder. 2020. http:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-first-drug-treat-rare-metabolic-disorder?utm_medium=email&utm_source=govdelivery. Accessed 25 Nov 2020."},{"key":"1463_CR7","unstructured":"European Medicines Agency. Oxlumo 94.5 mg\/0.5 mL, solution for injection: summary of product characteristics. 2020. http:\/\/ec.europa.eu\/health\/documents\/community-register\/2020\/20201119149780\/anx_149780_en.pdf. Accessed 24 Nov 2020."},{"key":"1463_CR8","unstructured":"Alnylam Pharmaceuticals. Oxlumo (lumasiran) injection, for subcutaneous use: US prescribing information. 2020. http:\/\/www.alnylam.com\/wp-content\/uploads\/pdfs\/OXLUMO-Prescribing-Information.pdf. Accessed 25 Nov 2020."},{"key":"1463_CR9","unstructured":"Alnylam Pharmaceuticals. Alnylam Pharmaceuticals and Taiba Group partner to commercialize RNAi therapeutics in the Gulf States [media release]. 8 July 2020. https:\/\/investors.alnylam.com\/press-release?id=24996."},{"key":"1463_CR10","unstructured":"Alnylam Pharmaceuticals, Dicerna Pharmaceuticals. Alnylam and Dicerna form RNAi therapeutics collaboration on alpha-1 antitrypsin deficiency-associated liver disease and complete cross-license agreement for primary hyperoxaluria programs [media release]. 6 Apr 2020. https:\/\/investors.alnylam.com\/press-release?id=24701."},{"key":"1463_CR11","unstructured":"Alnylam Pharmaceuticals. Alnylam Pharmaceuticals and Medison Pharma partner to commercialize RNAi therapeutics in Israel [media release]. 21 Jan 2019. https:\/\/www.businesswire.com\/news\/home\/20190121005029\/en\/Alnylam-Pharmaceuticals-and-Medison-Pharma-Partner-to-Commercialize-RNAi-Therapeutics-in-Israel."},{"key":"1463_CR12","doi-asserted-by":"crossref","unstructured":"Frishberg Y, Deschenes G, Groothoff J, et al. Final results from the phase 1\/2 trial of lumasiran and program updates. 2019. https:\/\/www.alnylam.com\/wp-content\/uploads\/2019\/06\/Luma_OHF-Ph12_Capella_FINAL.pdf. Accessed 14 Dec 2020.","DOI":"10.37940\/AJVS.2021.14.1.2"},{"key":"1463_CR13","doi-asserted-by":"crossref","unstructured":"Frishberg Y, Hulton S, Cochat P, et al. Results from the ongoing phase 2 open-label extension study of lumasiran, an investigational RNAi therapeutic, in patients with primary hyperoxaluria type 1 (PH1) [abstract no. PO1647 and presentation]. J Am Soc Nephrol. 2020;31(Abstract Suppl):520\u20131.","DOI":"10.1681\/ASN.20203110S1520d"},{"key":"1463_CR14","doi-asserted-by":"crossref","unstructured":"Saland J, Groothoff J, Frishberg Y, et al. 12-Month analysis of ILLUMINATE-A, a phase 3 study of lumasiran: sustained oxalate lowering and kidney stone event rates in primary hyperoxaluria type 1 [abstract no. PO2637 and presentation]. J Am Soc Nephrol. 2020;31(Abstract Suppl):B8.","DOI":"10.1681\/ASN.20203110S1B8c"},{"key":"1463_CR15","doi-asserted-by":"crossref","unstructured":"Michael M, Desch\u00eanes G, Cochat P, et al. ILLUMINATE-B, a phase 3 open-label study to evaluate lumasiran, an RNAi therapeutic, in young children with primary hyperoxaluria type 1 (PH1) [abstract no. PO1624 and presentation]. J Am Soc Nephrol. 2020;31(Abstract Suppl):515.","DOI":"10.1681\/ASN.20203110S1515a"},{"key":"1463_CR16","doi-asserted-by":"crossref","unstructured":"van't Hoff W, Cochat P, Groothoff J, et al. Safety and efficacy of lumasiran, an investigational RNA interference (RNAi) therapeutic, in adult and pediatric patients with primary hyperoxaluria type 1 [abstract no. SUN-325]. Kidney Internat Rep. 2019;4(7 Suppl):S295.","DOI":"10.1016\/j.ekir.2019.05.734"},{"key":"1463_CR17","doi-asserted-by":"crossref","unstructured":"Garrelfs S, Frishberg Y, Hulton S, et al. ILLUMINATE-A, A phase 3 study of lumasiran, an investigational RNAi therapeutic, in children and adults with primary hyperoxaluria type 1 (PH1) [abstract no. LB002 and presentation]. In: Nephrology Dialysis Transplantation. 2020.","DOI":"10.1093\/ndt\/gfaa146.LB002"}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-020-01463-0.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s40265-020-01463-0\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-020-01463-0.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,8,21]],"date-time":"2024-08-21T19:26:16Z","timestamp":1724268376000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/s40265-020-01463-0"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,1,6]]},"references-count":17,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2021,2]]}},"alternative-id":["1463"],"URL":"https:\/\/doi.org\/10.1007\/s40265-020-01463-0","relation":{},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,1,6]]},"assertion":[{"value":"6 January 2021","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. Lesley Scott and Susan Keam are salaried employees of Adis International Ltd\/Springer Nature, and declare 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"}}]}}