The U.S. Food and Drug Administration (FDA) has approved the use of tezacaftor/ivacaftor (Symdeko®) for children with cystic fibrosis ages 6 to 11 who have two copies of the F508del mutation or a single copy of one of 26 specified mutations. An additional 2,000 children will now qualify for the CFTR modulator, which was first approved in 2018 for people with CF ages 12 and older.
Tezacaftor/ivacaftor provides an alternative option for people with qualifying mutations, including individuals who cannot tolerate the CFTR modulator lumacaftor/ivacaftor (Orkambi ®).
The drug is a combination of two CFTR modulators which work in tandem to improve the function of the CFTR protein. It works by enabling the protein to form the right shape and allowing it to move to the surface of cells lining the lungs and other organs. With a functioning protein, chloride is then able to flow in and out of the cells, thereby reducing the symptoms of CF.
“We are pleased that more children with CF will have an additional modulator which will allow them to benefit from a treatment for the underlying cause of their disease,” said Michael P. Boyle, MD, senior vice president of therapeutic development for the Cystic Fibrosis Foundation. “This is an important step forward, as research has shown that taking modulators at a young age has the potential to slow or even prevent the progression of the disease.”
Tezacaftor/ivacaftor was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific, and financial support from the Cystic Fibrosis Foundation.
The Foundation is committed to finding treatments for the underlying cause of the disease for all people with CF, and significant programs are underway to develop treatments for people with nonsense and rare mutations that will not benefit from CFTR modulators as well as treatments for the complications of the disease. To learn more about the research the Foundation is supporting in these areas, please visit the Research We Fund section of cff.org.
For additional information regarding today's announcement, please see links to the press releases from the FDA and Vertex.