FDA Expands Symdeko Indications


Approved to treat pediatric patients ages 6 years and older with cystic fibrosis


Symdeko (a combination of tezacaftor/ivacaftor) tablets, from Vertex Pharmaceuticals, was approved to treat pediatric patients ages 6 years and older with cystic fibrosis who have certain genetic mutations. The new indications, announced today from the FDA, follow last year’s approval of the pharmaceutical drug to treat patients ages 12 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. 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,” explains 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 in a prepared statement.

“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,” Karimi-Shah says.

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Cystic fibrosis results in the formation of thick mucus that builds up in the lungs, digestive tract and other parts of the body, FDA reports. This inherited disease progresses to severe respiratory and digestive problems, and it can lead to infections and diabetes. The disease is caused by a defective protein that results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “There are approximately 2,000 known mutations of the CFTR gene. Symdeko is used to treat patients who have two copies of the most common type of mutation – F508del mutation – or who have at least one of the mutations in the CFTR gene that is responsive to the active ingredients in Symdeko based on in vitro data and/or clinical evidence, FDA reports.

The efficacy of Symdeko in patients with cystic fibrosis age 12 years and older was evaluated in three, Phase 3 double-blind, placebo-controlled trials, FDA says. Trial results demonstrated improvements in lung function and other key measures. The efficacy in patients ages 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 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 that had similar observations of safety to clinical trials in ages 12 and older, FDA states. The safety and efficacy of Symdeko in patients with cystic fibrosis younger than 6 years of age have not been studied, the agency adds.


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