{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T07:25:34Z","timestamp":1791444334917,"version":"4.3.3"},"reference-count":15,"publisher":"Springer Science and Business Media LLC","issue":"15","license":[{"start":{"date-parts":[[2021,9,30]],"date-time":"2021-09-30T00:00:00Z","timestamp":1632960000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2021,9,30]],"date-time":"2021-09-30T00:00:00Z","timestamp":1632960000000},"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":[[2021,10]]},"DOI":"10.1007\/s40265-021-01600-3","type":"journal-article","created":{"date-parts":[[2021,9,30]],"date-time":"2021-09-30T11:09:01Z","timestamp":1633000141000},"page":"1803-1809","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":34,"title":["Avalglucosidase alfa: First Approval"],"prefix":"10.1007","volume":"81","author":[{"given":"Sohita","family":"Dhillon","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,9,30]]},"reference":[{"issue":"13","key":"1600_CR1","doi-asserted-by":"publisher","first-page":"291","DOI":"10.21037\/atm.2019.04.15","volume":"7","author":"HV Do","year":"2019","unstructured":"Do HV, Khanna R, Gotschall R. Challenges in treating Pompe disease: an industry perspective. Ann Transl Med. 2019;7(13):291.","journal-title":"Ann Transl Med."},{"issue":"9","key":"1600_CR2","doi-asserted-by":"publisher","first-page":"1339","DOI":"10.3390\/biom10091339","volume":"10","author":"NK Meena","year":"2020","unstructured":"Meena NK, Raben N. Pompe disease: new developments in an old lysosomal storage disorder. Biomolecules. 2020;10(9):1339.","journal-title":"Biomolecules"},{"issue":"48","key":"1600_CR3","doi-asserted-by":"publisher","first-page":"50336","DOI":"10.1074\/jbc.M409676200","volume":"279","author":"Y Zhu","year":"2004","unstructured":"Zhu Y, Li X, Kyazike J, et al. Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice. J Biol Chem. 2004;279(48):50336\u201341.","journal-title":"J Biol Chem"},{"key":"1600_CR4","unstructured":"US FDA. FDA approves new treatment for Pompe Disease [media release]. 6 Aug 2021. https:\/\/www.fda.gov\/."},{"key":"1600_CR5","unstructured":"Genzyme Corp. Nexviazyme\u2122 (avalglucosidase alfa): US prescribing information. 2021. https:\/\/products.sanofi.us\/nexviazyme\/nexviazyme.pdf. Accessed 16 Aug 2021."},{"key":"1600_CR6","unstructured":"European Medicines Agency. Nexviadyme (avalglucosidase alfa): summary of opinion (initial authorisation). 2021. https:\/\/www.ema.europa.eu\/en\/documents\/smop-initial\/chmp-summary-positive-opinion-nexviadyme_en.pdf. Accessed 16 Aug 2021."},{"issue":"3","key":"1600_CR7","doi-asserted-by":"publisher","first-page":"167","DOI":"10.1016\/j.nmd.2018.12.004","volume":"29","author":"LDM Pena","year":"2019","unstructured":"Pena LDM, Barohn RJ, Byrne BJ, et al. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naive and alglucosidase alfa-treated patients with late-onset Pompe disease: a phase 1, open-label, multicenter, multinational, ascending dose study. Neuromuscul Disord. 2019;29(3):167\u201386.","journal-title":"Neuromuscul Disord"},{"issue":"6","key":"1600_CR8","doi-asserted-by":"publisher","first-page":"954","DOI":"10.1038\/mt.2009.37","volume":"17","author":"Y Zhu","year":"2009","unstructured":"Zhu Y, Jiang JL, Gumlaw NK, et al. Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease. Mol Ther. 2009;17(6):954\u201363.","journal-title":"Mol Ther"},{"issue":"Suppl 1","key":"1600_CR9","first-page":"326","volume":"26","author":"B Schoser","year":"2019","unstructured":"Schoser B, Barohn R, Dimachkie M, et al. NEO1 and NEO-EXT studies: pharmacodynamic and exploratory biomarker assessments following repeat avalglucosidase alfa dosing for up to 4.5 years in patients with late-onset Pompe disease [abstract no. EPR3085]. Eur J Neurol. 2019;26(Suppl 1):326.","journal-title":"Eur J Neurol"},{"key":"1600_CR10","first-page":"355","volume":"5","author":"V Straub","year":"2017","unstructured":"Straub V, Choi YC, Manera JD, et al. Comet methodology: comparison of the efficacy and safety of the enzyme replacement therapies, neogaa and alglucosidase alfa, in treatment-naive patients with late-onset Pompe disease [abstract no. 792]. J Inborn Errors Metab Screen. 2017;5:355\u20136.","journal-title":"J Inborn Errors Metab Screen."},{"issue":"2","key":"1600_CR11","first-page":"S57","volume":"132","author":"PS Kishnani","year":"2021","unstructured":"Kishnani PS, Attarian S, Borges JL, et al. Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in late-onset Pompe disease patients [abstract no. 121]. Mol Genet Metab. 2021;132(2):S57.","journal-title":"Mol Genet Metab"},{"issue":"2","key":"1600_CR12","doi-asserted-by":"publisher","first-page":"S34","DOI":"10.1016\/j.ymgme.2020.12.064","volume":"132","author":"MM Dimachkie","year":"2021","unstructured":"Dimachkie MM, Barohn RJ, Byrne B, et al. NEO1\/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset Pompe disease (LOPD) [abstract no. 58]. Mol Genet Metab. 2021;132(2):S34.","journal-title":"Mol Genet Metab"},{"issue":"Suppl 1","key":"1600_CR13","first-page":"333","volume":"28","author":"P Carlier","year":"2021","unstructured":"Carlier P, Vissing J, Diaz Manera J, et al. NEO-EXT: skeletal muscle % fat in late-onset Pompe disease after long-term avalglucosidase alfa (3-point Dixon MRI) [abstract no. EPR-189]. Eur J Neurol. 2021;28(Suppl 1):333.","journal-title":"Eur J Neurol"},{"issue":"2","key":"1600_CR14","first-page":"S57","volume":"132","author":"PS Kishnani","year":"2021","unstructured":"Kishnani PS, Brassier A, Broomfield A, et al. Mini-COMET study: individual participant-level responses to treatment in patients with infantile-onset Pompe disease receiving repeated dose regimens of avalglucosidase alfa or alglucosidase alfa who were previously treated with alglucosidase alfa [abstract no. 122]. Mol Genet Metab. 2021;132(2):S57\u20138.","journal-title":"Mol Genet Metab"},{"issue":"2","key":"1600_CR15","doi-asserted-by":"publisher","first-page":"S31","DOI":"10.1016\/j.ymgme.2020.12.058","volume":"132","author":"J Davison","year":"2021","unstructured":"Davison J, Brassier A, Broomfield A, et al. Mini-COMET study: effects of repeat avalglucosidase alfa dosing on ptosis in participants with infantile-onset Pompe disease (IOPD) who were previously treated with alglucosidase alfa [abstract no. 52]. Mol Genet Metab. 2021;132(2):S31\u20132.","journal-title":"Mol Genet Metab"}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-021-01600-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40265-021-01600-3\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-021-01600-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,10,26]],"date-time":"2021-10-26T15:59:01Z","timestamp":1635263941000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40265-021-01600-3"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,9,30]]},"references-count":15,"journal-issue":{"issue":"15","published-print":{"date-parts":[[2021,10]]}},"alternative-id":["1600"],"URL":"https:\/\/doi.org\/10.1007\/s40265-021-01600-3","relation":{},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,9,30]]},"assertion":[{"value":"30 September 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. Sohita Dhillon 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"}}]}}