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.
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 the US, the FDA has approved the expanded use of Kalydeco® for people with the following cystic fibrosis gene mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D and R117H.
In Australia, Kalydeco® is approved for reimbursement on the PBS for adults and children aged 6 and over.
The TGA has approved Kalydeco® in children 2-5 years. It is awaiting PBAC recommendation for reimbursement.
In the US, this drug was developed by Vertex Pharmaceuticals, Inc. with significant scientific, clinical and financial support from the Cystic Fibrosis Foundation Therapeutics (CFFT). The drug development was conducted within CFFT's Therapeutics Development Network.
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