Featured Research

from universities, journals, and other organizations

FDA approves new drug for cystic fibrosis

Date:
January 31, 2012
Source:
University of Alabama at Birmingham
Summary:
The FDA's approval of ivacaftor for treatment of cystic fibrosis will provide substantial benefit for CF patients with the G551D mutation in CFTR, say investigators who conducted clinical trials on the drug.

The U.S. Food and Drug Administration has approved the first drug to treat the underlying cause of cystic fibrosis, ivacaftor -- marketed under the name Kalydeco. Kalydeco is approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator gene.

"Today was a big day for the CF community," said Steven Rowe, M.D., MSPH, associate professor in the Division of Pulmonary, Allergy and Critical Care Medicine at the University of Alabama at Birmingham School of Medicine, who lead multiple studies of the new drug. "I believe this new therapy will substantially improve the lives of CF patients with the G551D mutation, and we hope that with appropriate testing it will also soon also be shown to help patients with other CFTR mutations as well."

The drug targets a defective chloride channel protein in patients with the G551D mutation, enabling that protein to function more efficiently.

Approximately 1,200 people in the United States, or 4 percent of those with CF, are believed to have the G551D mutation. In the studies, those who were treated with Kalydeco experienced significant and sustained improvements in lung function as well as other disease measures, including weight gain, frequency of hospitalization for CF respiratory problems, and certain quality-of-life measurements, compared to those who received placebo.

The majority of adverse events associated with Kalydeco were mild to moderate. Adverse events commonly observed included headache, upper respiratory tract infection or a common cold, stomach pain and diarrhea.


Story Source:

The above story is based on materials provided by University of Alabama at Birmingham. Note: Materials may be edited for content and length.


Cite This Page:

University of Alabama at Birmingham. "FDA approves new drug for cystic fibrosis." ScienceDaily. ScienceDaily, 31 January 2012. <www.sciencedaily.com/releases/2012/01/120131171816.htm>.
University of Alabama at Birmingham. (2012, January 31). FDA approves new drug for cystic fibrosis. ScienceDaily. Retrieved August 30, 2014 from www.sciencedaily.com/releases/2012/01/120131171816.htm
University of Alabama at Birmingham. "FDA approves new drug for cystic fibrosis." ScienceDaily. www.sciencedaily.com/releases/2012/01/120131171816.htm (accessed August 30, 2014).

Share This




More Health & Medicine News

Saturday, August 30, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

We've Got Mites Living In Our Faces And So Do You

We've Got Mites Living In Our Faces And So Do You

Newsy (Aug. 30, 2014) A new study suggests 100 percent of adult humans (those over 18 years of age) have Demodex mites living in their faces. Video provided by Newsy
Powered by NewsLook.com
Liberia Continues Fight Against Ebola

Liberia Continues Fight Against Ebola

AFP (Aug. 30, 2014) Authorities in Liberia try to stem the spread of the Ebola epidemic by raising awareness and setting up sanitation units for people to wash their hands. Duration: 00:41 Video provided by AFP
Powered by NewsLook.com
California Passes 'yes-Means-Yes' Campus Sexual Assault Bill

California Passes 'yes-Means-Yes' Campus Sexual Assault Bill

Reuters - US Online Video (Aug. 30, 2014) California lawmakers pass a bill requiring universities to adopt "affirmative consent" language in their definitions of consensual sex, part of a nationwide drive to curb sexual assault on campuses. Linda So reports. Video provided by Reuters
Powered by NewsLook.com
3 Things To Know About The Ebola Outbreak's Progression

3 Things To Know About The Ebola Outbreak's Progression

Newsy (Aug. 29, 2014) Here are three things you need to know about the deadly Ebola outbreak's progression this week. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins