Lumacaftor and ivacaftor (Orkambi®) is a combination therapy combining lumacaftor, which is designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface, with ivacaftor, which helps improve the function of the protein as a chloride channel on the cell surface.
In the US, the FDA has approved the use of Orkambi® in people with CF who have two copies of the F508del CFTR mutation and are 6 years and older. A phase 3 study of Orkambi® in children aged 2-5 years with two copies of the F508del CFTR mutation is currently underway.
In Australia, ORKAMBI is approved by PBAC and reommended for reimbursement from 1 October 2018
In the US, Vertex Pharmaceuticals developed this drug with funding from Cystic Fibrosis Foundation Therapeutics (CFFT). The drug development was conducted within CFFT's Therapeutics Development Network.
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