The FDA has approved Trikafta, a new triple combination therapy from Vertex Pharma, for the treatment of cystic fibrosis (CF) in patients 12 years of age and older who have at least one f508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Trikafta is comprised of elexacaftor/tezacaftor/ivacaftor and ivacaftor and is designed to increase the quantity and function of the f508del-CFTR protein at the cell surface, according to the company’s official release
The treatment will cost $311,501 annually, or $23,896 per 28-day pack.
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“Today marks a milestone for 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 U.S., TRIKAFTA is the first medicine that can treat the underlying cause of their disease,” says Jeffrey Leiden, MD, PhD, Vertex's Chairman, President and Chief Executive Officer in a statement. “I want to personally thank the hundreds of Vertex scientists who have been working on this program for nearly 20 years—many of whom have dedicated their entire careers to changing the course of this disease; the CF Foundation which has provided support, encouragement and help throughout the journey; and most importantly the thousands of patients, caregivers, doctors and advocates who have courageously and persistently worked side-by-side with us to get to where we are today.”
The approval stems from the positive results of two global, phase 3 studies: a 23-week study comprising 403 people with f508del mutation and one minimal function mutation (F/MF) and a 4-week phase 3 study in 107 people with two f508del mutations (F/F).
Potential serious side effects of the use of Trikafta include high liver enzymes in the blood and abnormality of the eye lens.
Adverse events reported throughout clinical trials of Trikafta include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.