(170) Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from an open-label extension study

Background: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in participants 12 years and older with cystic fibrosis (CF) and at least one F508del-CFTR allele in two pivotal Phase 3 trials. A 192-week Phase 3, open-label extension study is being conducted to assess long-term safety and efficacy in these participants. Here, we report results of the 144-week interim analysis (IA).

Methods: Participants are receiving ELX 200 mg once daily/TEZ 100 mg once daily/IVA 150 mg every 12 hours. The primary endpoint is safety and tolerability; secondary endpoints include absolute changes in percent predicted FEV₁ (ppFEV₁), sweat chloride, CF Questionnaire-Revised (CFQ-R) respiratory domain score, and body mass index from parent study baseline, and number of pulmonary exacerbations (PEx). Annualized rate of change in ppFEV₁ will be assessed as a pre-planned ad hoc analysis. Data collection for the 144-week IA will be based on the date the last participant reaches at least Week 144.

Results: 506 participants (F508del/minimal function [F/MF] genotypes, n=399; F508del/ F508del [F/F] genotype, n=107) were enrolled and dosed. The last patient visit for this 144-week IA will take place on March 28, 2022 with final data available in late April 2022. Results from primary and secondary endpoints and ad hoc analysis will be presented.

Conclusions: Pending

Acknowledgements: This work was funded by Vertex Pharmaceuticals Incorporated.

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