WASHINGTON (CBS Local) — A treatment that could benefit 90 percent of cystic fibrosis patients was approved Monday by the U.S. Food and Drug Administration.
The FDA approved a combination of three drugs Monday, called Trikafta, for patients 12 years and older with cystic fibrosis. Trikafta targets a mutation in the F508del gene, which is found in approximately 27,000 people in the United States.
Cystic fibrosis, a rare, progressive, life-threatening disease, results in the formation of thick mucus that builds up in the lungs, digestive tract and other parts of the body. It leads to severe respiratory and digestive problems as well as other complications such as infections and diabetes.
Through FDA approval today, we are making a landmark treatment available to 90% of cystic fibrosis patients 12 & older – some who previously had no options – and giving others in the community access to an additional effective therapy. https://t.co/bFMml425Hn pic.twitter.com/Rcdipx4WxL
— Dr. Ned Sharpless (@FDACommissioner) October 21, 2019
Cystic fibrosis is caused by a defective protein that results from mutations in the in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There is no cure.
Trikafta targets the defective CFTR protein and helps the protein made by the CFTR gene mutation function more effectively.
Because of it’s benefit to the cystic fibrosis community, the FDA reviewed and approved Trikafta in approximately three months, far ahead of the March 19, 2020 review goal date.
The approval of Trikafta was granted to Boston-based Vertex Pharmaceuticals Incorporated, which has priced Trikafta at $311,503 per year or $23,896 per 28-day pack, according to the Securities and Exchange Commission.