Skip to content
My WebMD Sign In, Sign Up

Children's Health

Font Size

First Drug to Target Cause of Cystic Fibrosis Approved

Kalydeco Helps 4% of Cystic Fibrosis Patients; Future Treatment May Help Others

Kalydeco Approval: Personalized Medicine for CF continued...

According to Reuters, Kalydeco will cost $294,000 per year. The company says it has assistance programs in place for eligible CF patients not covered by insurance.

Only about 30,000 Americans have cystic fibrosis. But it's likely that CFTR drugs will have uses far beyond CF. For example, the drugs may be able to help the mucus buildup that's a key factor in COPD, the fourth leading cause of death in the U.S. and in Europe. CFTR may also play a role in smoke-induced lung damage.

Vertex isn't the only company working on CFTR drugs. FoldRx Pharmaceuticals, owned by Pfizer, is working on compounds that combine the CFTR corrector and CFTR potentiator functions.

A CF drug called ataluren is under development by PTC Therapeutics. Ataluren targets CFTR mutations seen in about 10% of CF patients.

1 | 2

Today on WebMD

preschool age girl sitting at desk
look at my hand
woman with cleaning products
young boy with fever

worried kid
boy on father's shoulder
Child with red rash on cheeks
girl thinking

Child with adhd
rl with friends
Syringes and graph illustration
6-Week Challenges
Want to know more?
Build a Fitter Family Challenge – Get your crew motivated to move.
Feed Your Family Better Challenge - Tips and tricks to healthy up your diet.
Sleep Better Challenge - Snooze clues for the whole family.
I have read and agreed to WebMD's Privacy Policy.
Enter cell phone number
- -
Entering your cell phone number and pressing submit indicates you agree to receive text messages from WebMD related to this challenge. WebMD is utilizing a 3rd party vendor, CellTrust, to provide the messages. You can opt out at any time.
Standard text rates apply

WebMD Special Sections