Cystic Fibrosis Foundation Statement on FDA Approval of TRIKAFTA, the First Triple-Combination Therapy for the Most Common CF Mutation
Today’s FDA approval of TRIKAFTA, the highly effective and highly anticipated triple-combination CFTR modulator, marks the start of a new era in cystic fibrosis. This medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually benefit more than 90 percent of people with CF.
“We at the CF Foundation, and many in the CF community, have dreamed about this milestone for decades,” said Dr. Preston Campbell, president and CEO of the Cystic Fibrosis Foundation. “Throughout my decades as a clinician caring for people with CF, I’ve looked forward to the day when we would be able to dramatically transform the treatment of this disease. I’m deeply grateful to be able to say that day is here.”
“This is a moment to celebrate and to reflect on how working together, and against great odds, we have effectively transformed a genetic disease in a single generation, making CF the greatest story in medicine,” said Dr. Michael Boyle, senior vice president of therapeutics development of the Cystic Fibrosis Foundation, who will succeed Dr. Campbell as president and CEO as of January 1, 2020. “As we celebrate today, we also vow to intensify our focus on finding a therapy for every individual in our community who is still waiting for a breakthrough. We will not rest until every person with CF has a treatment for the underlying cause of their disease and, one day, a cure.”
TRIKAFTA is approved for people 12 years and older with CF who have at least one F508del mutation in the CFTR gene. In clinical trials, TRIKAFTA showed dramatic improvements in key measures of the disease, such as lung function, sweat chloride, and quality of life—results that may have a transformative effect on people’s day-to-day lives.
