Positive results were announced today from a late stage study of Trikafta in children between the ages of 6 and 11 with cystic fibrosis.  Vertex Pharmaceuticals Inc. completed their trial of the CFTR modulator in children who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.

The 24-week Phase 3 study, which was evaluating the safety and efficacy of Trikafta in this age group, showed that the drug was generally well tolerated, and the safety data were consistent with those observed in previous studies. Additionally, participants in the trial showed improvements in their lung function (FEV), sweat chloride, and body mass index.  

“Initiating modulator therapy at a younger age has the potential to dramatically alter the course of CF for many people with the disease,” said JP Clancy, vice president of clinical research for the Cystic Fibrosis Foundation. “We have seen the transformative effect that Trikafta can have for an older population, we are excited by today's announcement and the prospect of this drug benefiting children ages 6 through 11.”

Based on these results, Vertex plans to submit a supplemental New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020.

For additional information regarding the results of these clinical trials, please see the Vertex press release.