FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older – Drug Information Update

FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older

The U.S. Food and Drug Administration today expanded the indication for Symdeko (a combination of tezacaftor/ivacaftor) tablets for treatment of pediatric patients ages 6 years and older with cystic fibrosis who have certain genetic mutations. Last year, the FDA approved Symdeko to treat patients ages 12 and older who had the same specific genetic mutations.

The safety of Symdeko to treat cystic fibrosis patients age 6 to less than 12 years was supported by data from a study that included a 24-week, open-label treatment period with 70 cystic fibrosis patients ages 6 to less than 12, and had similar observations of safety to clinical trials in ages 12 and older. Symdeko should always be taken with food that contains fat and never in combination with certain antibiotics, seizure medicines, St. John’s wort, or food containing grapefruit or Seville oranges, as indicated on the label. The prescribing information for Symdeko includes warnings related to elevated enzymes in the liver (transaminases) in people taking Symdeko, for those who use inducers for another liver enzyme called Cytochrome P450 3A4 (CYP3A), and for the risk of cataracts in pediatric patients. Patients and their caregivers should speak with a health care professional about these risks and any medicines they take before starting Symdeko. The most common side effects include headache, nausea, sinus congestion and dizziness.

The safety and efficacy of Symdeko in patients with cystic fibrosis younger than 6 years of age have not been studied.

To learn more, please visit: Symdeko.