FDA News

FDA expands Symdeko indication for cystic fibrosis in children as young as 6

The FDA has expanded the indication for combination tezcaftor/ivacaftor tablets to include treatment of cystic fibrosis in children aged 6 years and older who have certain genetic mutations in the gene, according to an agency press release.

Before expanding the indication, tezcaftor/ivacaftor (Symdeko, Vertex Pharmaceuticals) was approved to treat cystic fibrosis only in children aged 12 and older who have two copies of the F508del mutation in the CFTR gene or who have at least one of the mutations in the CFTR gene that is responsive to the active ingredients in the drug based on in vitro data or clinical evidence.

Three phase 3, double-blind, placebo-controlled trials in children with cystic fibrosis aged 12 and older showed improvements in lung function and other key measures of the disease, including a reduction in exacerbations. The FDA based its decision to expand the indication to younger patients based on extrapolation of efficacy data in patients aged 12 and older to those aged 6 to 12, with additional support from data in patients aged 6 to 12, according to the FDA release.

The agency also stated that the safety of tezcaftor/ivacaftor in younger patients was supported by data from a study, which included a 24-week, open-label treatment period with 70 children with cystic fibrosis aged 6 to younger than 12, had similar observations of safety to clinical trials in children aged 12 and older.

“Decades ago, patients with cystic fibrosis were generally expected to live until 10 years of age, with few surviving into their teenage years. Since then, wide-ranging research on the disease resulted in more treatments for this debilitating disease that have extended life expectancy and improved quality of life for patients, but there is still no cure,” Banu Karimi-Shah, MD, acting deputy director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, said in the press release. “Based on their individual genetic makeup, individuals may respond differently to certain drugs, so it is important to provide a variety of options. Today’s approval of Symdeko for children as young as 6 years old provides an important treatment option for younger patients, and also provides more context on the safety and dosing specific to this population. The FDA remains committed to advancing novel treatment options for areas of unmet patient need, particularly in diseases impacting children.”

In the release, the FDA noted that tezcaftor/ivacaftor 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. Prescribing information also includes warnings related to elevated enzymes in the liver in people taking the drug, for those who use inducers for another liver enzyme (cytochrome P450 3A4) and for the risk for cataracts in pediatric patients.

The most common effects include headache, nausea, sinus congestion and dizziness.

The FDA also noted that the safety and efficacy of tezcaftor/ivacaftor in patients with cystic fibrosis younger than 6 have not been studied. – by Melissa Foster

The FDA has expanded the indication for combination tezcaftor/ivacaftor tablets to include treatment of cystic fibrosis in children aged 6 years and older who have certain genetic mutations in the gene, according to an agency press release.

Before expanding the indication, tezcaftor/ivacaftor (Symdeko, Vertex Pharmaceuticals) was approved to treat cystic fibrosis only in children aged 12 and older who have two copies of the F508del mutation in the CFTR gene or who have at least one of the mutations in the CFTR gene that is responsive to the active ingredients in the drug based on in vitro data or clinical evidence.

Three phase 3, double-blind, placebo-controlled trials in children with cystic fibrosis aged 12 and older showed improvements in lung function and other key measures of the disease, including a reduction in exacerbations. The FDA based its decision to expand the indication to younger patients based on extrapolation of efficacy data in patients aged 12 and older to those aged 6 to 12, with additional support from data in patients aged 6 to 12, according to the FDA release.

The agency also stated that the safety of tezcaftor/ivacaftor in younger patients was supported by data from a study, which included a 24-week, open-label treatment period with 70 children with cystic fibrosis aged 6 to younger than 12, had similar observations of safety to clinical trials in children aged 12 and older.

“Decades ago, patients with cystic fibrosis were generally expected to live until 10 years of age, with few surviving into their teenage years. Since then, wide-ranging research on the disease resulted in more treatments for this debilitating disease that have extended life expectancy and improved quality of life for patients, but there is still no cure,” Banu Karimi-Shah, MD, acting deputy director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, said in the press release. “Based on their individual genetic makeup, individuals may respond differently to certain drugs, so it is important to provide a variety of options. Today’s approval of Symdeko for children as young as 6 years old provides an important treatment option for younger patients, and also provides more context on the safety and dosing specific to this population. The FDA remains committed to advancing novel treatment options for areas of unmet patient need, particularly in diseases impacting children.”

In the release, the FDA noted that tezcaftor/ivacaftor 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. Prescribing information also includes warnings related to elevated enzymes in the liver in people taking the drug, for those who use inducers for another liver enzyme (cytochrome P450 3A4) and for the risk for cataracts in pediatric patients.

The most common effects include headache, nausea, sinus congestion and dizziness.

The FDA also noted that the safety and efficacy of tezcaftor/ivacaftor in patients with cystic fibrosis younger than 6 have not been studied. – by Melissa Foster