{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,20]],"date-time":"2026-05-20T12:07:50Z","timestamp":1779278870501,"version":"3.51.4"},"reference-count":17,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2020,10,22]],"date-time":"2020-10-22T00:00:00Z","timestamp":1603324800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["bpspubs.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacology Res &amp; Perspec"],"published-print":{"date-parts":[[2020,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Deflazacort (Emflaza) was approved in the United States in 2017 for the treatment of the Duchenne muscular dystrophy in patients aged 2\u00a0years and older. Several deflazacort metabolites were isolated and identified from rats, dogs, monkeys, and humans. Among them, 1\u00df,2\u00df\u2010epoxy\u20103\u00df\u2010hydroxy\u201021\u2010desacetyl deflazacort, referred to as Metabolite V, was reported to be one of the major circulating metabolites in humans. However, its quantitative distribution in plasma was not fully characterized. The objective of this study was to determine deflazacort plasma pharmacokinetics, metabolite profiles and their quantitative exposures in humans following a single oral dose. Six healthy male subjects were each administered a single oral dose of 60\u00a0mg [<jats:sup>14<\/jats:sup>C]\u2010deflazacort. Plasma and urine were collected and deflazacort metabolites in plasma were quantified by high performance liquid chromatography radio\u2010profiling followed by liquid chromatography\u2010mass spectrometry characterization. Metabolite V was isolated from urine and its structure was further confirmed by nuclear magnetic resonance analysis. These analyses demonstrated that deflazacort was not detectable in plasma; of the eight circulating deflazacort metabolites identified or characterized, the pharmacologically active metabolite 21\u2010desacetyl deflazacort and inactive metabolite 6\u00df\u2010hydroxy\u201021\u2010desacetyl deflazacort accounted for 25.0% and 32.9% of the 0\u201024\u00a0hours plasma total radioactivity, respectively, while Metabolite V, an epoxide species, was a minor circulating metabolite, representing only about 4.7% of the total plasma radioactivity.<\/jats:p>","DOI":"10.1002\/prp2.677","type":"journal-article","created":{"date-parts":[[2020,10,22]],"date-time":"2020-10-22T17:07:10Z","timestamp":1603386430000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Metabolite V, an epoxide species is a minor circulating metabolite in humans following a single oral dose of deflazacort"],"prefix":"10.1002","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4692-7499","authenticated-orcid":false,"given":"Ronald","family":"Kong","sequence":"first","affiliation":[{"name":"PTC Therapeutics, Inc. 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South Plainfield NJ USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joseph","family":"Colacino","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc. South Plainfield NJ USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Francesco","family":"Bibbiani","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc. South Plainfield NJ USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2020,10,22]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.neulet.2012.04.078"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/0960-8966(91)90039-U"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2014-2044"},{"key":"e_1_2_10_5_1","unstructured":"Emflaza Prescribing Information. 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