Article
Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adolescents and adults with cystic fibrosis and F508del-gating and F508del-residual function genotypes: results from an open-label extension study.
Annals of the American Thoracic Society - 1 Sept 2026
Chmiel James F, McBennett Kimberly, Quon Bradley S, Colombo Carla, De Wachter Elke, Fajac Isabelle, Mondejar-Lopez Pedro, Polineni Deepika, Robinson Philip, Sutharsan Sivagurunathan, Mall Marcus A, McKone Edward F, Ramsey Bonnie, Taylor-Cousar Jennifer L, Tullis Elizabeth, Suresh Nina E, Zhang Yaohua, Sosnay Patrick, Jennings Mark, Barry Peter J
Abstract excerpt
RATIONALE: 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 ≥ 12 years 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...
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