Kalydeco, Drug That Treats Root Cause Of Cystic Fibrosis, Approved By FDA

Kalydeco, Drug That Treats Root Cause Of Cystic Fibrosis, Approved By FDA

www.huffingtonpost.com

The first drug that treats the root cause of cystic fibrosis won approval Tuesday, offering a life-changing treatment for a handful of patients with the deadly illness and broader hope for thousands more patients with the inherited disease.

(visit the link for the full news article)


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This is a great step forward for those of us with CF and the G551D mutation, while the damage is already done in my case this will help so many people suffering around the world.

I only hope that the drug is affordable so everyone can have access to it and that it's able to get worldwide as soon as possible.

While the newly named Kalydeco (Vx-770) targets only a small percentage of CF suffers they are working hard on combating the Double F508 which is much more common in the CF population.

We were all expecting it to be approved in late April so it's fantastic that it had been approved sooner.

www.huffingtonpost.com
(visit the link for the full news article)

This is good news and I hope those in discomfort get the proper assistance with their illnesses. Also is this related to the DRACO vaccine?

reply to post by CheekyRob

This is a great step forward for those of us with CF and the G551D mutation, while the damage is already done in my case this will help so many people suffering around the world.

i'm sorry to hear this.
but you've got my (mental) support

but the article states the following

"Even though this drug isn't for the majority of people, it proves that you can look at the mistake in the genes and design a drug in a rational way that will fix the problem," said Dr. Drucy Borowitz of the State University of New York at Buffalo, where she directs the cystic fibrosis program..

and this little tidbit

Vertex executives said Kalydeco would cost $294,000 for a year's supply, placing it among the most expensive prescription drugs sold in the U.S. Specialty drugmakers are known to charge $300,000 or more for drugs that treat very small groups of patients.

allthought it also states this:

"The drug is priced for the value it will deliver to this very small group of patients," Vertex Executive Vice President Nancy Wysenski told analysts.

Wysenski said Vertex would provide the medicine for free to people with no insurance and household income of $150,000 or less. The company will also cover 30 percent of copay costs for select patients who have insurance

I lost my son to CF when he was 12.
Its heartbreaking to see those with
the illness suffer and go down
hill. I am so glad that a drug as
been approved to treat CF and
I hope it works well.

Great to see this.

I had a girlfriend when I was 18 who died from Cystic Fibrosis, anything that can help even the smallest percentage of sufferers is a massive step in the right direction. Heres hoping for continued success in the development of the treatment