{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T15:19:03Z","timestamp":1792509543708,"version":"4.5.0"},"reference-count":16,"publisher":"Springer Science and Business Media LLC","issue":"8","license":[{"start":{"date-parts":[[2022,5,31]],"date-time":"2022-05-31T00:00:00Z","timestamp":1653955200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2022,5,31]],"date-time":"2022-05-31T00:00:00Z","timestamp":1653955200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Drugs"],"published-print":{"date-parts":[[2022,6]]},"DOI":"10.1007\/s40265-022-01727-x","type":"journal-article","created":{"date-parts":[[2022,5,31]],"date-time":"2022-05-31T10:02:47Z","timestamp":1653991367000},"page":"941-947","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":29,"title":["Olipudase Alfa: First Approval"],"prefix":"10.1007","volume":"82","author":[{"given":"Susan J.","family":"Keam","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2022,5,31]]},"reference":[{"issue":"9","key":"1727_CR1","doi-asserted-by":"publisher","first-page":"967","DOI":"10.1038\/gim.2017.7","volume":"19","author":"MM McGovern","year":"2017","unstructured":"McGovern MM, Dionisi-Vici C, Giugliani R, et al. Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency. Genet Med. 2017;19(9):967\u201374.","journal-title":"Genet Med"},{"issue":"2","key":"1727_CR2","doi-asserted-by":"publisher","first-page":"98","DOI":"10.1016\/j.ymgme.2018.11.014","volume":"126","author":"M Wasserstein","year":"2019","unstructured":"Wasserstein M, Dionisi-Vici C, Giugliani R, et al. Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD). Mol Genet Metab. 2019;126(2):98\u2013105.","journal-title":"Mol Genet Metab"},{"key":"1727_CR3","unstructured":"Sanofi. Xenpozyme\u00ae(olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency [media release]. 28 Mar 2022. http:\/\/www.sanofi.com."},{"key":"1727_CR4","unstructured":"Sanofi. Olipudase alfa (XENPOZYME\u00ae) IV infusion: Japanese prescribing information. 2022. https:\/\/www.pmda.go.jp\/PmdaSearch\/iyakuDetail\/780069_39594A9D1022_1_01. Accessed 31 Mar 2022"},{"key":"1727_CR5","unstructured":"Sanofi. CHMP recommends approval of Xenpozyme\u00ae (olipudase alfa), the first and only treatment for ASMD [media release]. 20 May 2022. https:\/\/www.sanofi.com."},{"issue":"1\u20132","key":"1727_CR6","doi-asserted-by":"publisher","first-page":"245","DOI":"10.1016\/j.ymgme.2020.06.010","volume":"131","author":"BL Thurberg","year":"2020","unstructured":"Thurberg BL, Diaz GA, Lachmann RH, et al. Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment. Mol Genet Metab. 2020;131(1\u20132):245\u201352.","journal-title":"Mol Genet Metab"},{"issue":"1","key":"1727_CR7","doi-asserted-by":"publisher","first-page":"34","DOI":"10.1038\/gim.2015.24","volume":"18","author":"MM McGovern","year":"2016","unstructured":"McGovern MM, Wasserstein MP, Kirmse B, et al. Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency). Genet Med. 2016;18(1):34\u201340.","journal-title":"Genet Med"},{"issue":"2","key":"1727_CR8","doi-asserted-by":"publisher","first-page":"217","DOI":"10.1016\/j.ymgme.2014.07.005","volume":"114","author":"JM Murray","year":"2015","unstructured":"Murray JM, Thompson AM, Vitsky A, et al. Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency: the utility of animal models of disease in the toxicological evaluation of potential therapeutics. Mol Genet Metab. 2015;114(2):217\u201325.","journal-title":"Mol Genet Metab"},{"key":"1727_CR9","doi-asserted-by":"publisher","DOI":"10.1016\/j.gim.2022.03.021","author":"M Wasserstein","year":"2022","unstructured":"Wasserstein M, Lachmann R, Hollak C, et al. A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: one-year results. Genet Med. 2022. https:\/\/doi.org\/10.1016\/j.gim.2022.03.021.","journal-title":"Genet Med"},{"issue":"8","key":"1727_CR10","doi-asserted-by":"publisher","first-page":"1543","DOI":"10.1038\/s41436-021-01156-3","volume":"23","author":"GA Diaz","year":"2021","unstructured":"Diaz GA, Jones SA, Scarpa M, et al. One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency. Genet Med. 2021;23(8):1543\u201350.","journal-title":"Genet Med"},{"issue":"2","key":"1727_CR11","doi-asserted-by":"publisher","first-page":"S37","DOI":"10.1016\/j.ymgme.2021.11.082","volume":"135","author":"GA Diaz","year":"2022","unstructured":"Diaz GA, Giugliani R, Guffon N, et al. Continued improvement in pulmonary, visceral, biomarker and growth outcomes in children with chronic acid sphingomyelinase deficiency treated with olipudase alfa enzyme replacement therapy: 2-year results of ASCEND-Peds [abstract no. 67]. Mol Genet Metab. 2022;135(2):S37.","journal-title":"Mol Genet Metab"},{"issue":"2","key":"1727_CR12","doi-asserted-by":"publisher","first-page":"S70","DOI":"10.1016\/j.ymgme.2021.11.175","volume":"135","author":"R Lachmann","year":"2022","unstructured":"Lachmann R, Diaz GA, Wasserstein MP, et al. Sustained and continued improvements in pulmonary function, hepatosplenomegaly, dyslipidemia, and disease biomarkers in 5 adults with chronic acid sphingomyelinase deficiency after 6.5 years of olipudase alfa enzyme replacement therapy [abstract no. 160]. Mol Genet Metab. 2022;135(2):S70.","journal-title":"Mol Genet Metab"},{"issue":"1\u20132","key":"1727_CR13","doi-asserted-by":"publisher","first-page":"88","DOI":"10.1016\/j.ymgme.2015.05.013","volume":"116","author":"MP Wasserstein","year":"2015","unstructured":"Wasserstein MP, Jones SA, Soran H, et al. Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency. Mol Genet Metab. 2015;116(1\u20132):88\u201397.","journal-title":"Mol Genet Metab"},{"issue":"1","key":"1727_CR14","doi-asserted-by":"publisher","first-page":"107","DOI":"10.1186\/s13023-021-01739-y","volume":"16","author":"B Garside","year":"2021","unstructured":"Garside B, Ho JH, Kwok S, et al. Changes in PCSK 9 and apolipoprotein B100 in Niemann-Pick disease after enzyme replacement therapy with olipudase alfa. Orphanet J Rare Dis. 2021;16(1):107.","journal-title":"Orphanet J Rare Dis"},{"issue":"3","key":"1727_CR15","doi-asserted-by":"publisher","first-page":"S176","DOI":"10.1016\/j.gim.2022.01.315","volume":"24","author":"M Wasserstein","year":"2022","unstructured":"Wasserstein M, Barbato A, Gallagher R, et al. Continued improvement in adults with acid sphingomyelinase deficiency after 2 years of olipudase alfa in the ASCEND placebo-controlled trial [abstract eP280]. Genet Med. 2022;24(3):S176\u20137.","journal-title":"Genet Med"},{"issue":"5","key":"1727_CR16","doi-asserted-by":"publisher","first-page":"829","DOI":"10.1007\/s10545-017-0123-6","volume":"41","author":"MP Wasserstein","year":"2018","unstructured":"Wasserstein MP, Diaz GA, Lachmann RH, et al. Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months. J Inherit Metab Dis. 2018;41(5):829\u201338.","journal-title":"J Inherit Metab Dis"}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-022-01727-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40265-022-01727-x\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-022-01727-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,6,20]],"date-time":"2022-06-20T16:36:59Z","timestamp":1655743019000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40265-022-01727-x"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,5,31]]},"references-count":16,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2022,6]]}},"alternative-id":["1727"],"URL":"https:\/\/doi.org\/10.1007\/s40265-022-01727-x","relation":{},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,5,31]]},"assertion":[{"value":"31 May 2022","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 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"}}]}}