About ivacaftor<< Back to CFTR modulators approved
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In Europe and the USA, ivacaftor is a prescription medicine used for the treatment of CF in individuals who are aged 6 years and older, and have at least one copy of the CFTR mutation, G551D, or any of the following gating mutations: G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. In Canada, the same mutations apply in addition to G907R for the treatment of CF in individuals who are aged 6 years and older.
- Sustained improvements in lung function including a reduction in pulmonary exacerbations
- Weight gain
- Reduced hospitalisations
There were no significant safety signals identified in the pivotal trials and an ongoing study is assessing the long-term safety of ivacaftor.7