Drug Development Pipeline
Restore CFTR Function
Ivacaftor (Kalydeco®) is an oral medication that was the first drug available that targeted the underlying cause of CF – the defective CFTR protein. Ivacaftor helps facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.
Ivacaftor is approved for children as young as 4 months old who have at least one of 97 ivacaftor-responsive CFTR mutations (see page 7 for a list of approved mutations).
In clinical trials, people with CF who took the drug had improved lung function, reduced pulmonary exacerbations, increased weight and improved quality of life measures. Additionally, the safety profile of ivacaftor in very young children was similar to that observed in older children and adults.
This drug was developed by Vertex Pharmaceuticals, Inc. with significant scientific, clinical and financial support from the Cystic Fibrosis Foundation. The drug development was conducted within the Therapeutics Development Network.
Recent Ivacaftor (Kalydeco®) Studies
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