The U.S. Food and Drug Administration has approved Vertex’s Trikafta (elexacaftor/ tezacaftor/ ivacaftor and ivacaftor) for the treating persons, 12 years and older, with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This is the most common mutation that results in cystic fibrosis.
With this approval, 6,000 patients with cystic fibrosis who previously did not have a treatment option, will be eligible to receive Trikafta. In addition, up to 12,000 patients currently taking other treatments may also be eligible.
Cystic fibrosis is a rare genetic condition due to mutation in the CFTR gene that lead to a lack of the CFTR protein that disrupts the proper flow of salt and water in various organs, most notably in the lungs.
The approval of Trikafta was largely based on two global Phase III studies in people ages 12 years and older with cystic fibrosis: a 24-week Phase III study in 403 people with one F508del mutation and one minimal function mutation, and a 4-week Phase III study in 107 people with two F508del mutations.
In both trials, the primary endpoint was the percent predicted forced expiratory volume in one second (ppFEV1). In the first trial, the mean increase in ppFEV1 was 13.8% from baseline compared to placebo. In the second trial, the mean increase in ppFEV1 was 10% from baseline compared to tezacaftor/ivacaftor.
The most common adverse drug reactions were headaches, upper respiratory tract infections, abdominal pains, diarrhea, rashes, increased liver enzymes, nasal congestion, increased blood creatine phosphokinase, rhinitis, influenza, sinusitis and increased blood bilirubin.
In a news release, acting FDA Commissioner Ned Sharpless, MD, said, “Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy.”
Vertex’s Chairman, President and Chief Executive Office, Jeffrey Leiden, MD, PhD, echoed Dr. Sharpless assessment. ““Today marks a milestone for cystic fibrosis (CF) patients, their families and Vertex. After a 20-year journey together, we have received FDA approval of Trikafta: a single breakthrough medicine with the potential to treat up to 90% of all people with CF in the future. For approximately 6,000 people with CF in the US, Trikafta is the first medicine that can treat the underlying cause of their disease.”
