FDA Directs Vertex to Conduct Kalydeco Study to Assess Potential Eye Risk
August 29, 2012
The U.S. Food and Drug Administration (FDA) today notified the CF community and health care providers about a potential safety concern for cataract development in children with CF who are taking Kalydeco™.
The FDA has directed the manufacturer of Kalydeco, Vertex Pharmaceuticals Inc., to conduct a clinical study to assess the risk of cataracts in children who are on the drug. A cataract is a clouding of the lens in the eye that can affect vision.
The FDA’s action was based on recent results from a laboratory study of Kalydeco. In the study, cataracts were seen in juvenile rats treated with the drug. It is unknown whether this study is relevant to assessing the risk to children, since there are differences in eye development between humans and rats.
According to Vertex, there have been no reports of cataracts among children or adults with CF who are currently taking Kalydeco or among those who participated in the Phase 3 trials of the drug prior to its approval in January 2012.
Until further information is available, it is recommended that people with the G551D mutation of CF who are currently taking Kalydeco continue with their treatment.
People with CF who have questions about the FDA announcement should talk with their CF physician or health care provider.
- Read the FDA’s statement.
- People with CF and their families who have questions about Kalydeco can contact Vertex directly at 1-877-752-5933.