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.

In 30 seconds

This phase 3 open-label extension study evaluated the long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in 251 adolescents and adults with cystic fibrosis and F508del-gating or F508del-residual function genotypes. Results showed that 96.0% experienced at least one adverse event, primarily mild or moderate, while maintaining improvements in lung function and other health metrics over 96 weeks.

Key findings

• 96.0% of participants experienced at least one adverse event, mostly mild (32.3%) or moderate (55.0%).
• Participants maintained improvements in percent predicted FEV1, sweat chloride concentration, and CFQ-R respiratory domain score.
• In Part B, 73.8% of participants had at least one adverse event, with none leading to discontinuation.

Why it matters

The findings support the long-term safety and efficacy of ELX/TEZ/IVA in managing cystic fibrosis in adolescents and adults, highlighting its potential to improve lung function and overall health in this population.