In the United States, about 1,500 people with CF in this age group have one of these mutations.
“While we are disappointed about the decision, we understand the complexity of the evaluation and look forward to working with the
In its application to the FDA, Vertex Pharmaceuticals Inc. requested expanding the use of ivacaftor to people with one of 23
Vertex developed ivacaftor with significant clinical and financial support from the CF Foundation. It was the first drug to target the defective
The drug was previously approved by the FDA for people with CF ages 2 and older who have one of 10 other rare mutations of CF. Ivacaftor is now available to about 2,000 people with CF in the U.S.
Ivacaftor increases the activity of the CFTR protein on the
For more information, see the Vertex press release.
Learn more about ivacaftor.