• BREAKING NEWS ABC shows live and on-demand -- Download the WATCH ABC app!

Kalydeco approved for cystic fibrosis patients

March 28, 2013 12:00:00 AM PDT
Cystic fibrosis is a chronic disease that affects about 30,000 kids and adults in the U.S. but patients sometimes have to take up to 20 pills a day to manage it.

Each year, 1,000 children are born with cystic fibrosis. It's a difficult existence. Many take numerous medications and therapeutically beat their chests to break up thick mucus.

Kids with cystic fibrosis are not expected to live past their thirties, but now thanks to a new treatment many could live much longer.

Rylee, a 6-year-old living with cystic fibrosis, is a girl on the go. She won't let her disease slow her down.

Cystic fibrosis causes the body to produce a thick mucus that clogs the lungs and pancreas, making it difficult to clear the lungs and break down food. A child with cystic fibrosis constantly fights infections and finds it hard to maintain a healthy weight.

"Any kind of plan that we had for the future or dream that we had for our daughter was completely shattered," Rylee's mom, Amy, said.

A year ago, Rylee, was taking 20 pills a day. Now, she's only taking one.

Kalydeco is the first drug that targets the underlying cause of cystic fibrosis, the defective protein called cystic fibrosis transmembrane conductance regulator. In some patients, this protein does not allow fluids to clean the surface of the lungs that can cause deadly infections.

That infection can fester because it just doesnt move," Dr. Carolyn Cannon, MD, Director of the Pediatric Cystic Fibrosis Care and Teaching Center at Children's Medical Center at Dallas, said.

Kalydeco restores the function of the cystic fibrosis transmembrane conductance regulator protein, clearing out the lungs, and relieving symptoms of the disease.

"It is absolutely a game changer. It changes their life," Dr. Cannon said.

Not only does Kalydeco take away the symptoms for some kids with cystic fibrosis, doctors believe it might allow them to live decades longer than expected, giving kids like Rylee a chance for a long, healthy life.

"I can dream anything for her now," Amy said.

Kalydeco was recently approved by the FDA for kids over six. Rylee is using it off-label and is the first child under the age of six to start on it.

The drug costs $300,000 a year, but Rylee's insurance pays all but $90 a month.


Load Comments