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.

Related Articles


"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 December 19, 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 December 19, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Friday, December 19, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

The Best Tips to Curb Holiday Carbs

The Best Tips to Curb Holiday Carbs

Buzz60 (Dec. 19, 2014) It's hard to resist those delicious but fattening carbs we all crave during the winter months, but there are some ways to stay satisfied without consuming the extra calories. Vanessa Freeman (@VanessaFreeTV) has the details. Video provided by Buzz60
Powered by NewsLook.com
Sierra Leone Bikers Spread the Message to Fight Ebola

Sierra Leone Bikers Spread the Message to Fight Ebola

AFP (Dec. 19, 2014) More than 100 motorcyclists hit the road to spread awareness messages about Ebola. Nearly 7,000 people have now died from the virus, almost all of them in west Africa, according to the World Health Organization. Video provided by AFP
Powered by NewsLook.com
Researchers Test Colombian Village With High Alzheimer's Rates

Researchers Test Colombian Village With High Alzheimer's Rates

AFP (Dec. 19, 2014) In Yarumal, a village in N. Colombia, Alzheimer's has ravaged a disproportionately large number of families. A genetic "curse" that may pave the way for research on how to treat the disease that claims a new victim every four seconds. Duration: 02:42 Video provided by AFP
Powered by NewsLook.com
Double-Amputee Becomes First To Move Two Prosthetic Arms With His Mind

Double-Amputee Becomes First To Move Two Prosthetic Arms With His Mind

Buzz60 (Dec. 19, 2014) A double-amputee makes history by becoming the first person to wear and operate two prosthetic arms using only his mind. Jen Markham has the story. Video provided by Buzz60
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:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

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