Therapy Areas: Hereditary Disorders
The US FDA expands approved use of Kalydeco to treat additional mutations of cystic fibrosis to 33
18 May 2017 -

The US Food and Drug Administration, an agency within the US Department of Health and Human Services, said on Wednesday that it has expanded the approved use of Boston-based Vertex Pharmaceuticals Inc's Kalydeco (ivacaftor) for treating cystic fibrosis.

This US FDA approval triples the number of rare gene mutations that the drug can now treat, expanding the indication from the treatment of ten mutations, to 33. The agency based its decision, in part, on the results of laboratory testing, which it used in conjunction with evidence from earlier human clinical trials.

Cystic fibrosis affects the cells that produce mucus, sweat and digestive juices. These secreted fluids build up in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems, as well as other complications such as infections and diabetes.

In conjunction, the results from an in vitro cell-based model system have been shown to reasonably predict clinical response to Kalydeco. When additional mutations responded to Kalydeco in the laboratory test, researchers were thus able to extrapolate clinical benefit demonstrated in earlier clinical trials of other mutations. This resulted in the addition of gene mutations for which the drug is now indicated, stated the US FDA.

Kalydeco, available as tablets or oral granules taken two times a day with fat-containing food, helps the protein made by the CFTR gene function better and as a result, improves lung function and other aspects of cystic fibrosis, including weight gain, added the US FDA.