OBJECTIVES: ELX/TEZ/IVA is a breakthrough therapy targeting the underlying cause of cystic fibrosis that is currently not available in Brazil. ELX/TEZ/IVA has been shown to substantially improve lung function and nutritional outcomes. Although survival projections are available in non-Brazilian settings, the potential long-term impact of ELX/TEZ/IVA on Brazilian pwCF is of interest given regional variation in survival. Here we estimate survival in Brazilian F/MF pwCF aged ≥6 years receiving ELX/TEZ/IVA+best supportive care (BSC) versus BSC-alone.

METHODS: A validated lifetime person-level simulation model was used to estimate survival in F/MF pwCF aged ≥6 years (mean age, 20.1 years) in Brazil with and without ELX/TEZ/IVA+BSC treatment. Clinical efficacy inputs were derived from ELX/TEZ/IVA Phase 3 studies that included F/MF pwCF (NCT03691779, NCT03525444). Lifetime outcomes were also evaluated separately for cohorts initiating ELX/TEZ/IVA+BSC during ages 6-11 and ≥12 years to understand impacts of early treatment initiation. To evaluate nearer-term benefits of ELX/TEZ/IVA in F/MF pwCF aged ≥6 years, an alternative scenario was run using a 10-year time horizon.

RESULTS: ELX/TEZ/IVA+BSC was projected to increase median survival in F/MF pwCF aged ≥6 years by 35.4 years versus BSC-alone (67.6 versus 32.2 years). The incremental median predicted survival benefit increased to 42.1 years in the cohort initiating ELX/TEZ/IVA+BSC during ages 6-11 years (72.3 versus 30.2 years with BSC-alone). In the 10-year alternative scenario, 89% of pwCF receiving ELX/TEZ/IVA+BSC were projected to be alive 10 years post-treatment initiation versus 72% receiving BSC-alone.

CONCLUSIONS: Based on these simulations, ELX/TEZ/IVA+BSC is projected to substantially increase survival in Brazilian F/MF pwCF aged ≥6 years. Initiating treatment earlier leads to even greater projected survival benefits; model projections suggest that initiation during ages 6-11 years may add upwards of 4 decades of survival benefit versus BSC-alone, allowing achievement of near-normal life expectancy. Results should be confirmed with real-world data, when available.