Today marks a tremendous breakthrough and exciting news for people with cystic fibrosis. The U.S. Food and Drug Administration (FDA) approved Trikafta™ for people with cystic fibrosis who have at least one copy of the F508del mutation. With the approval of Trikafta, more than 90 percent of people with CF could eventually have a highly effective therapy for the underlying cause of their disease. Until now, most people with a single copy of the F508del mutation did not have an approved treatment for the underlying cause of CF.
“Today marks a tremendous breakthrough and exciting news for people with cystic fibrosis,” said Preston W. Campbell, III, M.D., president and CEO of the Cystic Fibrosis Foundation. “This milestone is the result of an extraordinary community working together against great odds, and we are overjoyed that this will mean more people will have effective treatments for their disease.”
Clinical trials of Trikafta™ showed dramatic improvements in key measures of the disease. People with two copies of the F508del mutation had a 10 percent increase in lung function compared to treatment with the modulator tezacaftor/ivacaftor (Symdeko®), and people with one copy of F508del had more than a 14 percent increase in lung function compared to placebo.
“As we celebrate today, we will not lose sight of the many individuals in our community who are still waiting for a breakthrough that will treat their mutations,” said Michael P. Boyle, M.D., senior vice president of therapeutics development. “We will not be done until every person with CF has a treatment for the underlying cause of their disease and, one day, a cure.”
Late stage clinical trials of Trikafta™ are underway for children with CF ages 6 to 11. Data from that trial is expected in 2020.
The drug comes with warnings related to elevated liver function tests, drug-drug interactions with products that are inducers or inhibitors of a certain liver enzyme, and the risk of cataracts.
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. Learn more about the potential treatments the Foundation is exploring for people with nonsense and other rare mutations.
For more information on this announcement, please see the FDA press release.