Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for 24 Weeks or Longer in People with Cystic Fibrosis and One or More <i>F508del</i> Alleles: Interim Results of an Open-Label Phase 3 Clinical Trial

Safety results from this interim analysis were consistent with the initial 24-week placebo-controlled F/MF pivotal study, with similar or lower exposure-adjusted event rates observed in the OLE (Table 1) (5). ELX/TEZ/IVA was generally safe and well tolerated. Most AEs were consistent with common manifestations of CF and were not treatment limiting (3, 10). In participants who received ELX/TEZ/IVA in parent studies, improvements in efficacy and PD measures, including ppFEV1, SwCl concentration, BMI, CFQ-R RD score, and PEx event rate, were maintained or continued to improve further over 24 weeks (F/MF genotypes) or 36 weeks (F/F genotype) of additional treatment. These results validate the durability of ELX/TEZ/IVA efficacy responses, with no emerging safety concerns. Among participants who had received placebo or TEZ/IVA in the respective parent studies, initiation of ELX/TEZ/IVA rapidly led to marked improvements in these efficacy measures that were consistent with the results seen in the ELX/TEZ/IVA arms of those parent studies. Thus, the results of this combined-group interim analysis demonstrate the safety and sustained efficacy of long-term ELX/TEZ/IVA treatment in pwCF 12 years old or older with one or more F508del alleles.