in patients aged 12 years or older, the first triple-combination therapy approved for that indication.
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Approval for Trikafta was based on results from two clinical trials in patients with cystic fibrosis with an F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In the first trial, a 24-week, randomized, double-blind, placebo-controlled study of 403 patients, the mean percent predicted forced expiratory volume in 1 second increased by 14% from baseline, compared with placebo. In the second trial, a 4-week, randomized, double-blind, active-controlled study of 107 patients, mean percent predicted forced expiratory volume in 1 second was increased 10% from baseline, compared with tezacaftor/ivacaftor, according to the FDA press release.
In the first trial, patients who received Trikafta also saw improvement in sweat chloride, reduction in the number of pulmonary exacerbations, and reduction of body mass index, compared with placebo.
The most common adverse events associated with Trikafta during the trials were headaches, upper respiratory tract infections, abdominal pains, diarrhea, rashes, and rhinorrhea, among others. The label includes a warning related to elevated liver function tests, use at the same time with products that induce or inhibit a liver enzyme called cytochrome P450 3A4, and cataract risk.
“At the FDA, we’re consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review. Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy,” said acting FDA Commissioner Ned Sharpless, MD.
Find the full press release on the FDA website.