The U.S. Food and Drug Administration (FDA) approved Kalydeco® (ivacaftor) for infants with cystic fibrosis ages 1 month to 4 months who have certain mutations. Although this approval affects a very small number of infants, it is another important step in making sure that everyone with CF has a treatment for the underlying cause of their disease.
Modulators, such as Kalydeco, partially restore activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which maintains the proper balance of salt and fluids at the surface of the lungs and other organs. Research has shown that pancreatic function can be restored in some young children who take Kalydeco.1
“We want people with CF to start treatment with modulators as early as possible, as research indicates that they can help slow or even prevent the irreversible progression of this disease and in some cases rescue pancreatic function,” said JP Clancy, MD, senior vice president of clinical research at the Cystic Fibrosis Foundation. “This is the youngest population ever to be approved for a modulator, and we believe that it could help transform the trajectory of their disease and reduce the need for future chronic therapies.”
Given the importance of early intervention, the Foundation continues to support programs and policies that improve cystic fibrosis newborn screening. In 2022, the Foundation — along with external subject matter experts, clinical partners, and parents of children living with CF — established a newborn screening initiative to assess and improve equity, sensitivity, and timeliness in CF newborn screening to achieve nationwide better (or best) practices and improve health outcomes for all. This is especially important for those who receive delayed diagnoses or are misdiagnosed because of their race or ethnicity.
Kalydeco was initially approved in 2012 for treatment of people ages 6 and older with the gating mutation G551D, who make up 4% of the nearly 40,000 people with CF in the U.S. Since then, approval had been expanded to those as young as 4 months with one of 97 mutations.
Kalydeco was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific, and financial support from the Cystic Fibrosis Foundation.
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.
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.