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FDA expands Kalydeco label based on in vitro data

May 18, 2017 12:23 AM UTC

FDA expanded the label of cystic fibrosis drug Kalydeco ivacaftor to include 23 new disease-causing mutations, based on results from an in vitro cell model system. The agency said the approach "provides a pathway for adding additional, rare mutations of the disease, based on laboratory data."

In a blog post, FDA said the approval signals to other sponsors that in vitro assay data could be used "in place of additional small clinical trials when seeking to expand to other population subsets," for drugs with good safety profiles to treat well-characterized diseases. The agency noted that the in vitro data also identified mutations that were not responsive to Kalydeco. ...