FDA approves Kalydeco to treat rare form of cystic fibrosis

Friday, February 3, 2012 01:46 PM

The FDA has approved Kalydeco (ivacaftor), manufactured by Vertex Pharmaceuticals, for the treatment of a rare form of cystic fibrosis in patients ages six and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.

Cystic fibrosis is a serious genetic disorder affecting the lungs and other organs that ultimately leads to an early death. Of the 30,000 people in the U.S. with the common form, 4% are believed to have the G551D mutation.

The FDA reviewed and approved Kalydeco in approximately three months under the agency’s priority review program that is designed to expedite drugs of possible significance. Kalydeco was approved ahead of the drug’s April 18, 2012, prescription user fee goal date and is designated as an orphan drug, which identifies the disease as affecting fewer than 200,000 people in the U.S.

“Kalydeco is an excellent example of the promise of personalized medicine—targeted drugs that treat patients with a specific genetic makeup,” said FDA commissioner Margaret A. Hamburg, M.D.

In patients with the G551D mutation, Kalydeco, a pill taken two times a day with fat-containing food, helps the protein made by the CFTR gene function better and as a result, improves lung function and other aspects of CF such as increasing weight gain.

Two 48-week, placebo-controlled clinical studies involving 213 patients, one in patients ages 12 and older and another in patients ages six to 11, were used to evaluate the safety and efficacy of Kalydeco in cystic fibrosis patients with the G551D mutation. In both studies, treatment with Kalydeco resulted in significant and sustained improvement in lung function.

Share:          
CLINICAL TRIAL RESOURCES

Search:

NEWS ONLINE ARCHIVE

Browse by:

CWWeekly

December 15

Cancer Treatment Centers of America chooses WCG as its exclusive IRB, sheds five local hospital IRBs

Five southern research groups form public-private network to help save time, money for clients with early-stage biologics

Already a subscriber?
Log in to your digital subscription.

Subscribe to CWWeekly.

The CenterWatch Monthly

December

Growing adoption of feasibility review committees
Early reports cite fewer amendments, improved cycle time

AMCs vying to better compete for industry trials
Working to conquer study start-up delays, IRB review process

Already a subscriber?
Log in to your digital subscription.

Purchase the October issue.

Subscribe to
The CenterWatch Monthly.

The CenterWatch Monthly

November

Private equity driving dynamic CRO growth
Enables CROs to pursue long-term strategies, while giving the space credibility

Baby boomers poised to reshape clinical trials industry
Millions of tech-savvy consumers already track their health, expect new treatments as they age

Already a subscriber?
Log in to your digital subscription.

Purchase the September issue.

Subscribe to
The CenterWatch Monthly.

JobWatch centerwatch.com/jobwatch

Featured Jobs