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U.S. Department of Health and Human Services


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FDA Notification to the Cystic Fibrosis Community on Kalydeco (ivacaftor)

[8-29-2012] The U.S. Food and Drug Administration (FDA) is notifying health care professionals, parents/caregivers, and patients in the cystic fibrosis (CF) community about a potential safety concern for cataract development in children with CF taking the medication Kalydeco (ivacaftor). A cataract is a clouding of the lens in the eye that can affect vision. This potential safety concern is based on recent results from an animal study in juvenile rats that was conducted to support the treatment of young children less than two years of age with ivacaftor.

To inform health care professionals, information about the animal study has been added to the Nonclinical ToxicologyAnimal Toxicology and/or Pharmacology (13.2) section of the Kalydeco physician label. Cataracts were seen in juvenile rats treated with ivacaftor from postnatal days 7 to 35 at dose levels of 10 mg/kg/day and higher (approximately one tenth the maximum recommended human dose). There is uncertainty about the relevance of this risk to children, since there are differences in eye development between humans and rats.

To better assess the potential risk of cataracts in children receiving Kalydeco, FDA is requiring the manufacturer, Vertex Pharmaceuticals, Inc., to conduct a clinical study in pediatric patients up to 11 years of age who are being treated with Kalydeco. Children up to 11 years of age are included in the study, as eye development is generally complete by that age. Patients will be followed for at least two years and will receive eye examinations from an ophthalmologist at six-month intervals to assess for both visual acuity and cataracts/eye opacities.

Kalydeco is approved for the treatment of CF in patients age six years and older who have the specific G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. In patients with the G551D mutation, Kalydeco helps the protein made by the CFTR gene function better and, as a result, improves lung function and other aspects of CF such as increasing weight gain.

Until further information about these findings is known, it is recommended that CF patients with a G551D mutation in the CFTR who are receiving Kalydeco continue treatment. However, parents/caregivers are also encouraged to discuss the benefits and risks of Kalydeco treatment with their child’s health care professional. FDA also encourages patients taking Kalydeco to become part of the Cystic Fibrosis Foundation’s Patient Registry.