{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T01:46:24Z","timestamp":1791942384132,"version":"4.3.4"},"reference-count":46,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,10,9]],"date-time":"2024-10-09T00:00:00Z","timestamp":1728432000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:label\/><jats:p>Pegunigalsidase alfa, a PEGylated \u03b1\u2010galactosidase A enzyme replacement therapy (ERT) for Fabry disease, has a longer plasma half\u2010life than other ERTs administered intravenously every 2\u2009weeks (E2W). BRIGHT (NCT03180840) was a phase III, open\u2010label study in adults with Fabry disease, previously treated with agalsidase alfa or beta E2W for \u22653\u2009years, who switched to 2\u2009mg\/kg pegunigalsidase alfa every 4\u2009weeks (E4W) for 52\u2009weeks. Primary objective assessed safety, including number of treatment\u2010emergent adverse events (TEAEs). Thirty patients were enrolled (24 males); 23 previously received agalsidase beta. Pegunigalsidase alfa plasma concentrations remained above the lower limit of quantification throughout the 4\u2010week dosing interval. Thirty\u2010three of 182 TEAEs (in 9 patients) were considered treatment\u2010related; all were mild\/moderate. No patients developed de novo anti\u2010drug antibodies (ADAs). In the efficacy analysis (<jats:italic>n<\/jats:italic>\u2009=\u200929), median (inter\u2010quartile range) eGFR change from baseline over 52\u2009weeks was \u22121.9 (\u22125.9; 1.8) mL\/min\/1.73\u2009m<jats:sup>2<\/jats:sup> (<jats:italic>n<\/jats:italic>\u2009=\u200928; males [<jats:italic>n<\/jats:italic>\u2009=\u200922]: \u20132.4 [\u22125.2; 3.2]; females [<jats:italic>n<\/jats:italic>\u2009=\u20096]: \u22120.7 [\u22129.2; 2.0]). Overall, median eGFR slope was \u20131.9 (\u22128.3; 1.9) mL\/min\/1.73\u2009m<jats:sup>2<\/jats:sup>\/year (ADA\u2010negative [<jats:italic>n<\/jats:italic>\u2009=\u200920]: \u22121.2 [\u22126.4; 2.6]; ADA\u2010positive [<jats:italic>n<\/jats:italic>\u2009=\u20099]: \u22128.4 [\u221211.6; \u20131.0]). Lyso\u2010Gb3 concentrations were low and stable in females, with a slight increase in males (9\/24 ADA\u2010positive). The BRIGHT study results suggest that 2\u2009mg\/kg pegunigalsidase alfa E4W is tolerated well in stable adult patients with Fabry disease. Due to the low number of patients in this study, more research is needed to demonstrate the effects of pegunigalsidase alfa given E4W. Further evidence, outside of this clinical trial, should be factored in for physicians to prolong the biweekly ERT intervals to E4W.<\/jats:p><\/jats:sec><jats:sec><jats:title>Take\u2010home message<\/jats:title><jats:p>Treatment with 2 mg\/kg pegunigalsidase alfa every 4 weeks could offer a new treatment option for patients with Fabry disease.<\/jats:p><\/jats:sec>","DOI":"10.1002\/jimd.12795","type":"journal-article","created":{"date-parts":[[2024,10,9]],"date-time":"2024-10-09T04:44:41Z","timestamp":1728449081000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["A phase <scp>III<\/scp>, open\u2010label clinical trial evaluating pegunigalsidase alfa administered every 4\u2009weeks in adults with Fabry disease previously treated with other enzyme replacement therapies"],"prefix":"10.1002","volume":"48","author":[{"given":"Myrl","family":"Holida","sequence":"first","affiliation":[{"name":"Division of Medical 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