The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children with cystic fibrosis ages 6 through 11 who have at least one copy of the F508del mutation. Trikafta was also approved for children ages 6 through 11 who have certain mutations in the CFTR gene that are responsive based on lab data. With this approval, approximately 1,500 children will be eligible for a CFTR modulator for the first time.
“This approval is wonderful news for our community, especially for the families of those young children with CF who can now benefit from a modulator for the first time,” said Michael P. Boyle, MD, president and CEO of the Cystic Fibrosis Foundation. “We have seen the transformative effect that Trikafta can have on older children and adults, and we are excited by the prospect that this medicine may slow or prevent the progression of the disease at an early age and stave off the most common complications of CF before they start.”
Highly effective CFTR modulators, such as Trikafta, are known to have a transformative effect on the health and well-being of many people with CF. For example, studies have shown that some young children taking Kalydeco® (ivacaftor) have had their pancreatic function restored.1 But these therapies do not reverse established lung damage. This is one of the reasons it is important to start on CFTR modulators at a young age. Research suggests that beginning treatment with a modulator early could help slow or even prevent the irreversible progression of cystic fibrosis, dramatically altering the course of disease over time.
The FDA approval was based on data from a 24-week Phase 3 study, which was evaluating the safety and efficacy of Trikafta in children ages 6 to 11 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 12 and older in December 2020.
For additional information, please see the Vertex press release.
Despite extraordinary progress in helping people with CF live longer and healthier lives, there is still critical work to be done to help all people living with this disease, including those who won't benefit from modulators such as Trikafta. The Cystic Fibrosis Foundation's $500 million Path to a Cure is focused on developing new treatments for the underlying cause of the disease and, one day, a cure. This initiative centers around three core strategies to address the underlying cause of CF: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing or replacing the underlying genetic mutation to address the root cause of CF.
1Nichols AL, Davies JC, Jones D, Carr SB. Restoration of exocrine pancreatic function in older children with cystic fibrosis on ivacaftor. Paediatr Respir Rev. 2020 Sep;35:99-102. doi: 10.1016/j.prrv.2020.04.003. Epub 2020 Apr 14. PMID: 32386958.
Munce D, Lim M, Akong K. Persistent recovery of pancreatic function in patients with cystic fibrosis after ivacaftor. Pediatr Pulmonol. 2020 Dec;55(12):3381-3383. doi: 10.1002/ppul.25065. Epub 2020 Oct 22. PMID: 32910556.
Aoyama BC, Mogayzel PJ. Ivacaftor for the treatment of cystic fibrosis in children under six years of age. Expert Rev Respir Med. 2020 Jun;14(6):547-557. doi: 10.1080/17476348.2020.1741352. Epub 2020 Mar 17. PMID: 32154747.