Dear Friends,
Today marks a tremendous breakthrough and exciting news for people with cystic fibrosis.
The FDA announced its approval of TRIKAFTA, a new, highly effective CFTR modulator therapy that treats the underlying cause of CF. TRIKAFTA was approved for people 12 and older who have at least one F508del mutation. The approval of TRIKAFTA means that more than 90 percent of people with CF could eventually have a highly effective treatment for the underlying cause of their disease.
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 and better health and longer life may result. Just over 50 years ago, CF was a story of no research and no treatments; today, it is one of the most amazing stories in medicine.
Clinical trials of TRIKAFTA showed dramatic improvements in key measures of the disease, including lung function, sweat chloride, and quality of life. Not everyone will have the same results, but for many individuals with CF, TRIKAFTA has the potential to significantly change the course of their disease.
As we celebrate today, I know all of us will not lose sight of the many individuals in our community who are still waiting for a breakthrough that will treat their mutations. 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.
Thank you for your outstanding dedication to this shared mission and your commitment to making today’s milestone possible.
The FDA’s press release announcing the approval can be found here. We will share a web story and further information shortly. The Cystic Fibrosis Foundation is also planning a webinar to answer questions from the community about the FDA approval of TRIKAFTA. Details will be forthcoming.
Thank you,
Preston Campbell, MD
President and CEO
Michael Boyle, MD
SVP, Therapeutics Development
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