FDA Extends Trikafta Approval to Even Younger Cystic Fibrosis Patients

The FDA expanded the indication for elexacaftor, tezacaftor, and ivacaftor (Trikafta) in cystic fibrosis to include even younger children, maker Vertex Pharmaceuticals announced.

The three-drug combination's expanded label now covers children ages 2 to 5 years who have cystic fibrosis and either a F508del mutation in the CFTR gene or a CFTR gene mutation that has been shown to be responsive to the triplet based on in vitro data.

FDA approval for this indication was supported by a phase III open-label trial that showed improvements in sweat chloride concentration and lung function in this young population after 24 weeks of treatment, with a safety profile consistent with that observed in older age groups.

"Early intervention with CFTR modulator therapies like Trikafta can offer the potential to improve the trajectory of [cystic fibrosis] lung disease," said trial investigator Jennifer Goralski, MD, of University of North Carolina School of Medicine in Chapel Hill, in a statement. "With this approval, we now have the ability to treat young children with Trikafta and can proactively address the underlying cause of their disease."

As of 2022, approximately 40,000 people in the U.S. are living with cystic fibrosis.

Based on the results of two randomized trials, the triplet combination first won approval in 2019 for adolescents and adults with susceptible CFTR gene mutations, a group representing an estimated 90% of the population of patients with cystic fibrosis at that time. A subsequent label expansion allowed for use in patients ages 6 years and up as well.

Labeling for elexacaftor, tezacaftor, and ivacaftor warns of elevated transaminases and hepatic injury and cataracts. Additionally, concomitant use with strong CYP3A inducers may decrease the triple drug's exposure and efficacy.

The product is available in oral granules or tablets, depending on the patient's age and weight, and should be taken with fat-containing food. Patients are advised to avoid food or drink containing grapefruit while taking the triplet therapy.

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