FDA Expands Approval of Treatment for Cystic Fibrosis to Include Patients Aged 6 Years and Older

June 22, 2019

The US Food and Drug Administration (FDA) has expanded the indication for tezacaftor/ivacaftor tablets (Symdeko) for the treatment of paediatric patients aged 6 years and older with cystic fibrosis who have certain genetic mutations.

Last year, the FDA approved tezacaftor/ivacaftor to treat patients aged 12 years and older who had the same specific genetic mutations.

“Decades ago, patients with cystic fibrosis were generally expected to live until 10 years of age, with few surviving into their teenage years,” said Banu Karimi-Shah, MD, FDA’s Center for Drug Evaluation and Research, Rockville, Maryland. “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. 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 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,” he added. “The FDA remains committed to advancing novel treatment options for areas of unmet patient need, particularly in diseases impacting children.”

Tezacaftor/ivacaftor is used to treat patients who have 2 copies of the most common type of mutation -- F508del mutation -- or who have at least 1 of the mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor and ivacaftor based on in vitro data and/or clinical evidence.

The efficacy of tezacaftor/ivacaftor in patients with cystic fibrosis aged 12 years and older was evaluated in 3 phase 3, double blind, placebo-controlled trials, which demonstrated improvements in lung function and other key measures of the disease, including a reduction in exacerbations. The efficacy in patients aged 6 to 12 was extrapolated from patients age 12 years and older, with additional support from data in patients age 6 to 12 years.

The safety of tezacaftor/ivacaftor to treat patients aged 6 years to less than 12 years was supported by data from a study that included a 24-week, open-label treatment period with 70 patients and had similar observations of safety to clinical trials in patients aged 12 years and older.

Tezacaftor/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, as indicated on the label.

The prescribing information for tezacaftor/ivacaftor includes warnings related to elevated liver enzymes for those who use inducers for Cytochrome P450 3A4 (CYP3A), and for the risk of cataracts in paediatric patients.

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

Reference: shorturl.at/nHLTX

SOURCE: US Food and Drug Administration