New cystic fibrosis drug therapy seen as major breakthrough in treatment

A new drug therapy is showing remarkable results in patients with cystic fibrosis, according to a new study, alleviating symptoms and attacking the genetic root of the disease.

>> Read more trending news 

Researchers say that the new drug combination could benefit up to 90 percent of those who have the disease, something that has not been seen before, according to a story from the Washington Post.

The information was released Thursday at a national conference and was published in two medical journals. In a rare move, the Post reported, the Food and Drug Administration approved the three-drug combination which is called Trikafta, five months ahead of the agency's deadline.

Cystic fibrosis causes a buildup of thick mucus in the lungs and other organs and often kills those with the disease when they are in their teens.

The Cystic Fibrosis Foundation called the FDA's approval a "tremendous breakthrough" for patients. Around 30,000 people in the United States have cystic fibrosis, according to the CF Foundation.

Around half of those people are age 18 or older.

Francis Collins, the director of the National Institutes of Health, said the discovery was a "cause for celebration." Collins was part of the team that 30 years ago discovered the gene that mutates and causes the disease.

Dr. Jeffrey Leiden, the chairman of Vertex Pharmaceuticals, said the drug therapy "corrects the function of a mutant protein" that causes the disease.

The new drug therapy comes with a high price tag. According to Vertex, the therapy will cost $311,000 a year.

Vertex has a patient assistance program, called Vertex GPS, according to Cystic Fibrosis News Today. The program offers help to eligible patients in the U.S. who have been prescribed the medication.

Comments on this article