Wednesday, October 19, 2011


From the Cystic Fibrosis Foundation website

Vertex Submits Application to FDA for Approval of VX-770 – First Potential Drug to Target Underlying Cause of Cystic Fibrosis

October 19, 2011

Vertex Pharmaceuticals, Inc., announced today it has submitted an application to the U.S. Food and Drug Administration for a potential new CF therapy, VX-770 — under its new proposed trade name, KALYDECO™.
If approved, it will be the first drug on the market that targets the underlying cause of cystic fibrosis. Therapies available to people with CF to date only treat symptoms of the disease.
The company is seeking approval for the drug in people with cystic fibrosis age 6 and older who carry at least one copy of the G551D mutation of cystic fibrosis. (THAT'S ME!!!)
KALYDECO (kuh-LYE-deh-koh) was discovered in a collaboration between Vertex and the Cystic Fibrosis Foundation, which provided substantial scientific, financial and clinical support throughout the development process.
“The CF Foundation is thrilled that KALYDECO is on track for possible FDA approval in 2012,” said Robert J. Beall, Ph.D., President and CEO of the CF Foundation. “This is a significant step forward in our collaboration with Vertex and is further validation of the CF Foundation’s drug development strategy. We remain committed to accelerating the development of similar targeted medicines that will benefit all people with cystic fibrosis.”
Vertex has asked the FDA for priority review of the potential drug, which, if granted, could shorten the review from 10 to 6 months. The FDA grants priority review status for several reasons, including in situations where a potential drug is considered a major treatment advance. (YAY!!!!!)
Results released earlier this year from Phase 3 clinical trials of KALYDECO in people with the G551D mutation of CF showed that those receiving the drug had remarkable and sustained improvements in lung function and other key symptoms of the disease, compared with those on placebo. (YAY AGAIN!!!)
As FDA review of the potential drug gets underway, Vertex has set up a program to provide KALYDECO to people age 6 and older with the G551D mutation who are in critical medical need and could benefit from the treatment prior to potential approval. (Not me, but great idea for those who could benefit!)
The expanded access program is designed for people with CF who have highly limited lung function and meet other criteria. (Information about the program is available at CF Foundation-accredited care centers.)
KALYDECO is currently being evaluated in combination with another oral drug in development, VX-809, in people with the most common mutation of CF, Delta F508. 
Vertex plans to begin the second part of the Phase 2 KALYDECO and VX-809 clinical trial this month and will evaluate the two drugs over a longer period of time.

Frequently asked questions about Kalydeco (this one was my favorite):

What were the results of the VX-770 Phase 3 clinical trial in adults?
The Phase 3 clinical trial tested VX-770 in patients age 12 and older who carry at least one copy of the G551D mutation of the CF gene. Patients who received VX-770, compared to those on placebo, showed a marked improvement in lung function (FEV1).
Those who received the drug gained 10.6 percentage points more on a lung function test after 24 weeks than those getting a placebo, a difference that is statistically highly significant. Patients continued to take either drug or placebo for another 24 weeks and the improvement was sustained. Lung function, the primary endpoint of the trial, was measured by how much a person could exhale in one second, a standard test.
In addition, patients receiving VX-770 gained nearly seven pounds, on average, over the course of the trial. People with CF have a hard time gaining and maintaining weight because the buildup of mucus in the pancreas limits the body’s ability to absorb essential nutrients and vitamins.
Patients also showed improvement in other secondary endpoints of the study, including reduced likelihood of pulmonary exacerbation and decreased respiratory symptoms.
In addition, average sweat chloride dropped toward normal levels in patients on VX-770, compared to those on placebo. Excessive sweat chloride is a key clinical indicator of the disease.
The overall findings are profound because they demonstrate that a chemical compound can improve multiple clinical measures of CF by targeting the basic defect. CF therapies currently on the market address the symptoms of the disease, not the underlying cause.

This is such amazing news for me and everyone in the CF world!! Especially for me and the other 4% of the the CF population with the G551D mutation!!! The results of the clinical trail were outstanding!!! The progress of this drug gives me so much hope for the future. Yes, it may take another year or two before it is available for CF patients, but hey, I've been waiting 23 years for something like this, so what's another two?! I am very excited to share this news with my readers, friends and family. I am also thankful for your love and support over the years. Because of your donations, drugs like this are making significant progress and significant changes in peoples' lives. THANK YOU to anyone/everyone who has donated to the Cystic Fibrosis Foundation because you are making it possible for me to live a wonderful, happy, and healthy life.

I believe that one day this drug will help me achieve my ultimate goal: 
To LIVE, LAUGH, LOVE and BREATHE for a long time!!!!


  1. I am so happy that you are able to benefit from this drug! :) I'm waiting for the second one, but still I'm glad it's working it's way into our world! This is the best news in a long time! :)

  2. Yay! As jealous as I am (having DDF508)it is truly amazing how much closer we are getting to allowing CFers to lead a "normal" life. This will be such a profound impact on your life! I am really happy for you and all G551Ds!

  3. This is fantastic news. You are 4% in the CF world but #1 in the family.

    From Kirk and Lisa

  4. Thanks Kirk & Lisa, you guys are so sweet. :)

  5. Hi Colleen,

    This news on Kalydeco is travelling fast through the CF communities in Canada as well. My son Luke has CF (17yrs old) and news like this gives us REAL hope that life WILL get better for him and thousands of others living with this disease.

    Shari (Niagara Falls, Canada)