FDA Does Not Approve Ivacaftor for Some Rare Mutations of Cystic Fibrosis

The U.S. Food and Drug Administration (FDA) has decided not to approve the use of ivacaftor (Kalydeco®) to treat people with cystic fibrosis ages 2 and older who have one of 23 rare CF mutations, known as “residual function” mutations. 

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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 FDA as it continues to assess data and ensure that all drugs are safe and effective for patients,” said Preston W. Campbell III, M.D., president and CEO of the Cystic Fibrosis Foundation. “Our hope is that all people with CF who can benefit from the treatment will have the opportunity to do so.”

In its application to the FDA, Vertex Pharmaceuticals Inc. requested expanding the use of ivacaftor to people with one of 23 residual function mutations. Many of these mutations have only been evaluated for their responsiveness to ivacaftor in laboratory testing; late-stage clinical trials in patients with these mutations are ongoing. 

Vertex developed ivacaftor with significant clinical and financial support from the CF Foundation. It was the first drug to target the defective CFTR protein caused by mutations in the CF gene.

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 cell surface, which helps thin the thick mucus that builds up in the lungs of people with CF.

For more information, see the Vertex press release

Learn more about ivacaftor.

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