{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T23:46:07Z","timestamp":1791589567832,"version":"4.3.4"},"reference-count":27,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2024,10,7]],"date-time":"2024-10-07T00:00:00Z","timestamp":1728259200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008322","name":"CSL Behring","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008322","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Allergy"],"published-print":{"date-parts":[[2025,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Hereditary angioedema (HAE) is a chronic, unpredictable disease. Long\u2010term prophylactic treatments that offer durable efficacy, safety, and convenience are required to assist patients in achieving complete disease control, per international guidelines. We report an interim analysis of an ongoing phase 3 (VANGUARD) open\u2010label extension (OLE) study evaluating the long\u2010term safety and efficacy of garadacimab for HAE prophylaxis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Adults and adolescents aged \u226512\u2009years with HAE previously participating in phase 2 and pivotal phase 3 (VANGUARD) studies were rolled over to an OLE, alongside newly enrolled patients. Patients received garadacimab 200\u2009mg subcutaneously, once monthly for \u226512\u2009months. The primary endpoint was treatment\u2010emergent adverse events (TEAEs) in patients with C1 inhibitor deficiency\/dysfunction.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>At data cut\u2010off (February 13, 2023; <jats:italic>N<\/jats:italic>\u2009=\u2009161), median (interquartile range) exposure was 13.8\u2009months (11.9\u201316.3). For the primary endpoint, 133\/159 patients experienced \u22651 TEAE (524 events), equivalent to 0.23 events\/administration and 2.84 events\/patient\u2010year. Garadacimab\u2010related TEAEs (13% of patients, 52 events) were most commonly injection\u2010site reactions (ISRs). No deaths occurred. One patient discontinued treatment due to garadacimab\u2010related moderate ISR. Most TEAEs were mild\/moderate; three events were serious (COVID\u201019, two events; abdominal HAE attack, one event) and not garadacimab related. No abnormal bleeding, thromboembolic, severe hypersensitivity, or anaphylactic events were observed. Mean HAE attack rate decreased by 95% from the run\u2010in period; 60% of patients were attack\u2010free. Almost all patients (93%) rated their response to garadacimab as \u201cgood\u201d or \u201cexcellent.\u201d<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Garadacimab has a favorable safety profile suitable for long\u2010term use and provides durable protection against HAE attacks.<\/jats:p><\/jats:sec>","DOI":"10.1111\/all.16351","type":"journal-article","created":{"date-parts":[[2024,10,7]],"date-time":"2024-10-07T08:33:13Z","timestamp":1728289993000},"page":"545-556","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":33,"title":["Long\u2010term safety and efficacy of garadacimab for preventing hereditary angioedema attacks: Phase 3 open\u2010label extension\u00a0study"],"prefix":"10.1111","volume":"80","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3324-7072","authenticated-orcid":false,"given":"Avner","family":"Reshef","sequence":"first","affiliation":[{"name":"Allergy, Immunology and Angioedema Center Barzilai University Hospital  Ashkelon Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8951-8903","authenticated-orcid":false,"given":"Connie","family":"Hsu","sequence":"additional","affiliation":[{"name":"Research Solutions of Arizona  Litchfield Park Arizona USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4412-1336","authenticated-orcid":false,"given":"Constance H.","family":"Katelaris","sequence":"additional","affiliation":[{"name":"Allergy and Immunology Unit Campbelltown Hospital and Western Sydney University  Sydney New South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9155-9162","authenticated-orcid":false,"given":"Philip H.","family":"Li","sequence":"additional","affiliation":[{"name":"Division of Rheumatology and Clinical Immunology, Department of Medicine The University of Hong Kong  Pok Fu Lam Hong Kong"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9218-5468","authenticated-orcid":false,"given":"Markus","family":"Magerl","sequence":"additional","affiliation":[{"name":"Institute of Allergology Charit\u00e9 \u2013 University Medicine Berlin (joint medical faculty of Free University of Berlin and Humboldt University of Berlin)  Berlin Germany"},{"name":"Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology  Berlin Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9099-7673","authenticated-orcid":false,"given":"Keiko","family":"Yamagami","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine Osaka City General Hospital  Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7242-9584","authenticated-orcid":false,"given":"Mar","family":"Guilarte","sequence":"additional","affiliation":[{"name":"Allergology Department Vall d'Hebron University Hospital  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5644-1855","authenticated-orcid":false,"given":"Paul K.","family":"Keith","sequence":"additional","affiliation":[{"name":"McMaster University Medical Centre  Hamilton Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3476-1196","authenticated-orcid":false,"given":"Jonathan A.","family":"Bernstein","sequence":"additional","affiliation":[{"name":"Division of Rheumatology, Allergy and Immunology, Department of Internal Medicine University of Cincinnati  Cincinnati Ohio USA"},{"name":"Bernstein Clinical Research Center  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4321-176X","authenticated-orcid":false,"given":"John\u2010Philip","family":"Lawo","sequence":"additional","affiliation":[{"name":"CSL Innovation GmbH  Marburg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Harsha","family":"Shetty","sequence":"additional","affiliation":[{"name":"CSL Behring  King of Prussia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maressa","family":"Pollen","sequence":"additional","affiliation":[{"name":"CSL Behring  King of Prussia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lolis","family":"Wieman","sequence":"additional","affiliation":[{"name":"CSL Behring  King of Prussia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7774-4855","authenticated-orcid":false,"given":"Tim 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