Kalydeco Now Approved for Expanded Cystic Fibrosis Treatment

FDA officials have announced the expanded approval of ivacaftor (Kalydeco, Vertex Pharmaceuticals) to triple the number of rare gene mutations of cystic fibrosis that the drug is indicated to treat, from 10 mutations to 33.

The FDA based its approval on results of laboratory testing in conjunction with evidence from previous human clinical trials. Researchers identified additional mutations that responded to Kalydeco in testing and were able to extrapolate that data demonstrated in earlier clinical trials.

Kalydeco is available as tablets or oral granules and is to be taken 2 times a day with fat-containing food. It is designed to help the protein made by the

gene function better and improve lung function and other aspects of cystic fibrosis.

CFTR

According to an FDA press release, the expanded indication will affect another 3% of the cystic fibrosis population.

Adverse events that have been associated with this treatment include headache, upper respiratory tract infection, stomach pain, diarrhea, rash, nausea, and dizziness.

Reference

FDA expands approved use of Kalydeco to treat additional mutations of cystic fibrosis [news release]. FDA’s website.

. Accessed May 18, 2017.

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm559212.htm