The U.S. Food and Drug Administration (FDA) has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children with cystic fibrosis ages 2 through 5 years who have at least one copy of the F508del mutation or certain mutations that are responsive to Trikafta based on lab data. With this approval, approximately 2,250 children will be eligible for Trikafta, including more than 900 who will have access to a CFTR modulator for the first time.
“This approval is wonderful news for many families of young children with CF, whether they will now be able to receive a modulator for the first time or will be switching to a more effective therapy,” said Michael P. Boyle, MD, president and CEO of the Cystic Fibrosis Foundation. “The FDA’s decision gives additional momentum to our community’s ongoing efforts to bring effective treatments to more people with CF at the earliest age possible.”
CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that result in a dysfunctional CFTR protein. Modulators partially restore activity of the protein, which maintains the proper balance of salt and fluids at the surface of the lungs and other organs. By restoring the protein’s activity, many downstream consequences of CF could be prevented, reducing the need for future chronic therapies.
The FDA approval was based on data from a Phase 3 study, which evaluated the safety and efficacy of Trikafta in children ages 2 to 5 years with at least one F508del mutation. The study showed that the drug was generally well tolerated, and the safety data were consistent with those observed in previous studies. The FDA also approved Trikafta for children who have one of 177 other mutations that have been studied in the laboratory. The same laboratory data helped form the basis for approval of Trikafta for those ages 6 years and older.
Despite extraordinary progress in helping people with CF live longer and healthier lives, some people are either not eligible to take a modulator or they cannot tolerate the medication. There is still critical work to be done to help all people living with CF get a treatment for the underlying cause of their disease. To learn more about how we are funding research to find treatments for all people with CF, regardless of their mutations, visit Path to a Cure.