{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,27]],"date-time":"2026-08-27T21:17:30Z","timestamp":1787865450660,"version":"build-2784847793"},"reference-count":31,"publisher":"Oxford University Press (OUP)","issue":"9","license":[{"start":{"date-parts":[[2026,3,16]],"date-time":"2026-03-16T00:00:00Z","timestamp":1773619200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"Vertex Pharmaceuticals Incorporated"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,9,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Rationale<\/jats:title>\n                    <jats:p>In an 8-week, active-controlled, phase 3 study (445-104), elexacaftor\/tezacaftor\/ivacaftor (ELX\/TEZ\/IVA) was shown to be efficacious and safe in participants\u2009\u2265\u200912\u2009years of age with cystic fibrosis (CF) and F508del-gating (F\/G) or F508del-residual function (F\/RF) genotypes, conferring additional clinical benefits relative to IVA and TEZ\/IVA. Participants who completed this 8-week parent study continued to an open-label extension.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To assess the long-term safety and efficacy of ELX\/TEZ\/IVA in adolescents and adults with CF and F\/G or F\/RF genotypes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This 2-part (Part A [96\u2009weeks] and Part B [48\u2009weeks]) phase 3 open-label extension study enrolled adolescents and adults age\u2009\u2265\u200912\u2009years with CF and F\/G or F\/RF genotypes who completed study 445-104. Primary endpoint (Part A and Part B) was safety and tolerability. Secondary endpoints (Part A only) included absolute changes in percent predicted FEV1 (ppFEV1), sweat chloride concentration, CF Questionnaire-Revised (CFQ-R) respiratory domain score, and body mass index (BMI).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>A total of 251 participants received\u2009\u2265\u20091 dose of ELX\/TEZ\/IVA in Part A; 217 (86.5%) completed treatment. In Part A, 96.0% of participants had\u2009\u2265\u20091 adverse event (AE), which for most were mild (32.3%) or moderate (55.0%) in severity. Thirteen participants (5.2%) discontinued due to treatment-emergent AEs. Participants who received ELX\/TEZ\/IVA in the parent study maintained improvements in ppFEV1, sweat chloride concentration, CFQ-R respiratory domain score, and BMI while participants who received IVA or TEZ\/IVA (active controls) in parent study had similar improvements after transitioning to ELX\/TEZ\/IVA. Eighty-four participants (33.5%) entered Part B, 96.4% of whom discontinued due to commercial drug availability. In Part B, 62 participants (73.8%) had\u2009\u2265\u20091 AE, which for most were mild or moderate in severity and none of which led to discontinuation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>ELX\/TEZ\/IVA remained generally safe and well-tolerated with no new safety findings. Improvements in lung function, CFTR function, respiratory symptoms, and nutritional status after starting ELX\/TEZ\/IVA were maintained through 96\u2009weeks of follow-up. These results demonstrate the safety and durable efficacy of ELX\/TEZ\/IVA in adolescents and adults with F\/G or F\/RF genotypes.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/annalsats\/aaoag053","type":"journal-article","created":{"date-parts":[[2026,3,16]],"date-time":"2026-03-16T15:18:09Z","timestamp":1773674289000},"page":"1286-1295","source":"Crossref","is-referenced-by-count":0,"title":["Safety and efficacy of elexacaftor\/tezacaftor\/ivacaftor in adolescents and adults with cystic fibrosis and\n                    <i>F508del<\/i>\n                    -gating and\n                    <i>F508del<\/i>\n                    -residual function genotypes: results from an open-label extension study"],"prefix":"10.1093","volume":"23","author":[{"given":"James F","family":"Chmiel","sequence":"first","affiliation":[{"name":"Indiana University School of Medicine, Riley Hospital for Children , Indianapolis, IN,","place":["United 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