{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T10:02:05Z","timestamp":1753869725087,"version":"3.41.2"},"reference-count":34,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2025,6,13]],"date-time":"2025-06-13T00:00:00Z","timestamp":1749772800000},"content-version":"vor","delay-in-days":12,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100031034","name":"ISU Abxis","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100031034","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Molec Gen &amp;amp; Gen Med"],"published-print":{"date-parts":[[2025,6]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Imiglucerase (Cerezyme; Sanofi, Paris, France), an analogue of \u03b2\u2010glucocerebrosidase produced by recombinant DNA technology, has been a safe and effective treatment for Gaucher disease (GD) for over 25\u2009years. A new imiglucerase, Abcertin (Seongnam\u2010si, Gyeonggi\u2010do, Republic of Korea) has shown a similar safety and efficacy profile in previous clinical studies. This study compared the pharmacokinetics, immunogenicity, safety, and tolerability to EU\u2010sourced Cerezyme following a single 60\u2009IU\/kg dose.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This phase 1, single\u2010center, randomized, double\u2010blind, two\u2010way crossover study enrolled 36 healthy volunteers aged 18\u201345\u2009years. Participants were randomly assigned to receive either Abcertin or Cerezyme in a predetermined sequence.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Abcertin reached peak plasma concentrations at a median <jats:italic>t<\/jats:italic><jats:sub>max<\/jats:sub> of 61\u2009min (range: 40\u2013121\u2009min). The mean <jats:italic>C<\/jats:italic><jats:sub>max<\/jats:sub>, AUC<jats:sub>0\u2013last<\/jats:sub>, and AUC<jats:sub>0\u2013inf<\/jats:sub> were 115.4\u2009mU\/mL, 12,190\u2009min\u00b7mU\/mL, and 12,210\u2009min\u2009mU\/mL, respectively, indicating bioequivalence to Cerezyme. The mean <jats:italic>t<\/jats:italic><jats:sub>\u00bd<\/jats:sub>, CL, and <jats:italic>V<\/jats:italic><jats:sub>z<\/jats:sub> were 6.88\u2009min, 376.7\u2009mL\/min, and 3.62\u2009L, respectively, and were comparable between the two treatments. One participant in the Cerezyme group developed anti\u2010drug antibodies, which were non\u2010neutralizing A total of 24 subjects experienced treatment\u2010emergent adverse event (TEAE). The most common TEAE was headache (3 in the Abcertin group and 5 in the Cerezyme group), followed by general disorders and administration site condition (3 in Abcertin group and 5 in Cerezyme group). Two participants in the Cerezyme sequence experienced severe TEAEs: one had a urinary tract infection, and the other developed urticaria, which leading to study withdrawal.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Abcertin demonstrated pharmacokinetic equivalence to Cerezyme, with a comparable safety, immunogenicity, and tolerability profile. These findings support its potential as an affordable biosimilar for GD treatment.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mgg3.70111","type":"journal-article","created":{"date-parts":[[2025,6,14]],"date-time":"2025-06-14T06:54:01Z","timestamp":1749884041000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["A Randomized, Double\u2010Blind, 2\u2010Treatment, 2\u2010Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60\u2009<scp>IU<\/scp>\/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration"],"prefix":"10.1002","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6544-3599","authenticated-orcid":false,"given":"Eungu","family":"Kang","sequence":"first","affiliation":[{"name":"Department of Pediatrics Korea University Ansan Hospital, Korea University College of Medicine  Ansan Republic of Korea"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dohyung","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Asan Medical Center Children's Hospital, University of Ulsan College of Medicine  Seoul Republic of Korea"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Soojin","family":"Hwang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Asan Medical Center Children's Hospital, University of Ulsan College of Medicine  Seoul Republic of Korea"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Charlotte","family":"Lemech","sequence":"additional","affiliation":[{"name":"Scientia Clinical Research (SCR)  Randwick New South Wales Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jessica","family":"Wharton","sequence":"additional","affiliation":[{"name":"Scientia Clinical Research (SCR)  Randwick New South Wales Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yongyoon","family":"Lee","sequence":"additional","affiliation":[{"name":"ISU ABXIS Co., Ltd.  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