Drug Development Pipeline
Lumacaftor + ivacaftor (Orkambi®)
Restore CFTR Function
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.
The FDA has approved the use of Orkambi® in people with CF who have two copies of the F508del CFTR mutation and are 2 years and older.
A phase 3 study of Orkambi® in children ages 12 months to less than 24 months is underway.
Vertex Pharmaceuticals developed this drug with funding from the Cystic Fibrosis Foundation. The drug development was conducted within the Therapeutics Development Network.
Recent Lumacaftor + ivacaftor (Orkambi®) Studies
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