FDA approves Trikafta for children aged 2 to 5 years with cystic fibrosis

The FDA has expanded use of elexacaftor/tezacaftor/ivacaftor for children aged 2 to 5 years with cystic fibrosis with certain mutations, according to a press release from Vertex Pharmaceuticals.

This new approval applies to children in this age range who possess at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that responds to elexacaftor/tezacaftor/ivacaftor (Trikafta, Vertex Pharmaceuticals) according to in vitro data, according to the release.

In June 2021, this triple therapy gained FDA approval for children aged 6 to 11 with cystic fibrosis (CF) and the same mutations outlined above. Prior to that action, the FDA approved it for individuals aged 12 years and older with CF.

The new approval was based on a phase 3, open-label, multicenter trial that evaluated safety and tolerability of the therapy over 24 weeks among 75 children aged 2 to 5 years with one or more F508del mutation or an elexacaftor/tezacaftor/ivacaftor-responsive CFTR mutation.

As Healio previously reported, the study found that the children receiving the triple therapy tolerated it well and safety data was comparable to that of older children treated with the therapy. Additionally, improved measures of sweat chloride concentration and lung function were found with the triple therapy.

“Early intervention with CFTR modulator therapies like Trikafta can offer the potential to improve the trajectory of CF lung disease,” Jennifer Goralski, MD, assistant professor of medicine and pediatrics, co-director of the adult cystic fibrosis center at the University of North Carolina School of Medicine, and a lead principal investigator of the mentioned clinical trial, said in the release. “With this approval, we now have the ability to treat young children with Trikafta and can proactively address the underlying cause of their disease.”