FDA approves new ‘breakthrough’ drug for cystic fibrosis

National News

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.

Copyright 2020 Nexstar Broadcasting, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

Recent Videos

KX Storm Team Full Late Evening Forecast w/Tom Schrader 6/5

Thumbnail for the video titled "KX Storm Team Full Late Evening Forecast w/Tom Schrader 6/5"

Fastpitch Softball

Thumbnail for the video titled "Fastpitch Softball"

Babe Ruth Baseball

Thumbnail for the video titled "Babe Ruth Baseball"

OneFargo Peaceful Gathering

Thumbnail for the video titled "OneFargo Peaceful Gathering"

Friday, June 5th, 2020 - KX Storm Team Evening Forecast - Dave Holder

Thumbnail for the video titled "Friday, June 5th, 2020 - KX Storm Team Evening Forecast - Dave Holder"

Virtual Pet Adoption

Thumbnail for the video titled "Virtual Pet Adoption"

Cannabis Signature Drive

Thumbnail for the video titled "Cannabis Signature Drive"

Honor Flights

Thumbnail for the video titled "Honor Flights"

Sugar Spinning Sisters

Thumbnail for the video titled "Sugar Spinning Sisters"

SUPERSIZED KX Storm Team #OneMinuteForecast w/Tom Schrader 6/5

Thumbnail for the video titled "SUPERSIZED KX Storm Team #OneMinuteForecast w/Tom Schrader 6/5"

Amber's Friday Morning #OneMinuteForecast 6/5

Thumbnail for the video titled "Amber's Friday Morning #OneMinuteForecast 6/5"

Severe storms highlight the weekend forecast

Thumbnail for the video titled "Severe storms highlight the weekend forecast"

FURRY FRIDAY: MEET LADY DOGGA THE DOG

Thumbnail for the video titled "FURRY FRIDAY: MEET LADY DOGGA THE DOG"

LIFE HACKS: CARS

Thumbnail for the video titled "LIFE HACKS: CARS"

Beulah Baseball

Thumbnail for the video titled "Beulah Baseball"

Babe Ruth Baseball

Thumbnail for the video titled "Babe Ruth Baseball"

KX News Town-Hall 6/4

Thumbnail for the video titled "KX News Town-Hall 6/4"

Stress and Dreams

Thumbnail for the video titled "Stress and Dreams"

99th Birthday

Thumbnail for the video titled "99th Birthday"

Dickinson Parade

Thumbnail for the video titled "Dickinson Parade"
More Video

KX News Trending Stories

Don't Miss

Daily Pledge

More Daily Pledge