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# Vertex Expanded Use for Cystic Fibrosis Drug
- URL: https://www.fdaweb.com/vertex-expanded-use-for-cystic-fibrosis-drug/
- Published: 2018-08-07T12:00:00.000Z
- Updated: 2026-09-15T00:24:07.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5142058

FDA has approved an expanded use for Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to include use in children ages two through five years with cystic fibrosis (CF) who have two copies of the *F508del-CFTR* mutation, making it the first therapy approved to treat the underlying cause of CF in this population. Approval is based on data from a Phase 3 open-label safety study in 60 patients that showed treatment with Orkambi was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients ages six years and older, according to the company. Improvements in sweat chloride, a secondary endpoint, were observed at week 24, it says.

  
Orkambi was originally approved in 2015 to treat cystic fibrosis patients age 12 and older who have the *F508del* mutation that causes the production of an abnormal protein that disrupts how water and chloride are transported in the body. An FDA [statement](http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm453565.htm?ref=fdaweb.com) at the time said that having two copies of this mutation—one inherited from each patient—is the leading cause of cystic fibrosis.