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 ages 1 and older who have at least one of 38 ivacaftor-responsive CFTR 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.
In December 2017, a phase 3 trial for children ages 1 to 2 showed that ivacaftor was generally well tolerated, and that there were no new safety concerns in this population. Two phase 3 studies of ivacaftor in infants ages 0 to 2 are ongoing.
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
Latest News on Ivacaftor
August 15, 2018
Today, the U.S. Food and Drug Administration approved the use of ivacaftor (Kalydeco®) for children with cystic fibrosis ages 1 to 2 who have at least one mutation that is responsive to ivacaftor.
August 01, 2017
The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
May 17, 2017
The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
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