Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and indicated that it will make a decision by June 8, 2021.
In October 2019, the FDA approved Trikafta for people with CF ages 12 and older who have at least one F508del mutation. In December 2020, the FDA expanded approval to include individuals who are 12 years and older who have at least one of 177 additional mutations. Clinical trials of Trikafta showed dramatic improvements in lung function.
Research1 shows that starting on modulators at a young age could help slow or potentially prevent progression of the disease. Highly effective modulators, such as Trikafta, are known to have a transformative effect on the health and wellbeing of individuals eligible to take them.
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. Through its Path to a Cure, the Cystic Fibrosis Foundation intends to allocate $500 million to develop 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.