FDA approves new ‘breakthrough’ drug for cystic fibrosis

WASHINGTON (WCMH) — The U.S. Food and Drug Administration recently approved new medications to treat patients with cystic fibrosis.

Trikafta, (elexacaftor/ivacaftor/tezacaftor) is the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation, according to the FDA.

The drug was approved on Wednesday for patients 12 years and older with cystic fibrosis who have at least one F508del mutation, which is estimated to affect 90%, or 27,000, patients with the lung disease in the United States.

“Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy,” said acting FDA Commissioner Dr. Ned Sharpless. “In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients’ quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options.”

Acting FDA Commissioner Dr. Ned Sharpless.

Cystic fibrosis is a rare, life-threatening disease that 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.

Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene. While there are approximately 2,000 known mutations of the CFTR gene, the most common mutation is the F508del mutation.

Trikafta is a combination of three drugs that target the defective CFTR protein, according to the FDA.

It helps the protein made by the CFTR gene mutation function more effectively. Currently available therapies that target the defective protein are treatment options for some patients with cystic fibrosis, but many patients have mutations that are ineligible for treatment.

Trikafta has undergone two major clinical trials. In the results, patients saw improvements in lung function and body mass index.