FDA expands its approval of Trikafta to children ages 2 to 5

Expanded label could affect 900 children with CF in the US

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

Share this article:

Share article via email
Illustration of the word

The U.S. Food and Drug Administration (FDA) has approved the expanded use of Trikafta (elexacaftor, tezacaftor, and ivacaftor) to children with cystic fibrosis (CF) ages 2 to 5, who have at least one F508del mutation in the CFTR gene or a mutation that responds to Trikafta in laboratory studies.

Marketed by Vertex Pharmaceuticals, Trikafta was first approved by the FDA in 2019 for CF patients 12 and older with at least one F508del mutation, the most common mutation that causes CF. That approval has since been expanded to cover more CF-related mutations and children 6 and older.

“With this approval, we now have the ability to treat young children with TRIKAFTA and can proactively address the underlying cause of their disease,” Jennifer Goralski, MD, assistant professor of medicine and pediatrics, and co-director of the Adult Cystic Fibrosis Center at the University of North Carolina School of Medicine, said in a press release.

Recommended Reading
An illustration of a person's airways.

Trikafta rapidly improves lung function in real-world study

900 children with CF will now have access to Trikafta in US

The new expansion will allow, for the first time, about 900 children with CF to have a medicine to treat the underlying cause of their disease, according to Vertex.

This label expansion was supported by data from a Phase 3 clinical trial (NCT04537793) in children ages 2 to 5 with at least one F508del mutation in CFTR. Results showed that Trikafta was generally well-tolerated, with a safety profile similar to what was observed in older age groups. It was also effective in reducing sweat chloride concentration, a measure of CFTR function, and lung function. Body mass index, a measure of body fat, remained stable throughout the 24 weeks of treatment.

“Early intervention with CFTR modulator therapies like TRIKAFTA can offer the potential to improve the trajectory of CF lung disease,” said Goralski, who was also a lead principal investigator in the key clinical trial.

CF is caused by mutations in the CFTR gene that lead to a defective or missing CFTR protein. Children with CF inherit two defective CFTR gene copies, one from each parent.

Trikafta is an oral combination of three CFTR modulators — medications that work to improve the functionality of the mutated CFTR protein — called elexacaftor, tezacaftor, and ivacaftor.

“Since its initial approval, TRIKAFTA has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis,” said Carmen Bozic, MD, executive vice president of global medicines development and medical affairs, and chief medical officer at Vertex.

“We remain steadfast in our commitment to bringing highly effective CF treatments to people of all ages living with this disease,” she added.

Outside the U.S., Trikafta (or Kaftrio, the treatment’s name in certain countries) is approved for CF patients 6 and older with specific mutations in areas such as Canada, European Union, the U.K., Switzerland, Australia, and Israel.

Vertex is currently awaiting regulatory decisions in the E.U. and the U.K. for approval of the medication for children ages 2 to 5.