FDA Grants Six-Month Priority Review of Kalydeco (VX-770) — First Potential Drug to Target Underlying Cause of Cystic Fibrosis

Just a few days ago, I wrote a post about my annoyance with the FDA drug approval process when I heard that a "hangover pill" had made it through the pipeline while life saving medications like VX-770 sit in the queue. Today, my attitude toward them is changed. Today, Vertex Pharmaceuticals announced that the FDA has granted a six-month priority review for Kalydeco (vx-770)!!! The application for approval of the drug was made in October, which means that by April a decision will be made on the approval of this drug. By April, this drug could be available to us and begin correcting the problems that CF is causing at the cellular level. This is so incredible and awesome for so many reasons. We don't know if the drug will work for us, but we're going to give it a shot. If this drug works for Drew and he can get it before he has any progression of lung disease, his life expectancy will be the same as that of you or I. This drug will help his body to work normally, and prevent him from catching those life threatening lung infections that we deal with all to often. I'm so excited by this news and I hope that we can try it as soon as its approved...and that it works for us!!!

Here's the press release from the the Cystic Fibrosis Foundation:

FDA Grants Six-Month Priority Review of Kalydeco (VX-770) — First Potential Drug to Target Underlying Cause of Cystic Fibrosis

December 15, 2011

Vertex Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted a request for a six-month priority review of a potential new CF therapy, Kalydeco™ (VX-770).

The company is seeking approval of the drug for people ages 6 and older with the G551D mutation of CF.

The expedited review sets a target date of April 18, 2012, for the FDA’s approval decision, four months earlier than the standard review time of 10 months.

If approved, Kalydeco (kuh-LYE-deh-koh) will be the first drug available that targets the underlying cause of CF. The FDA grants priority review for several reasons, including situations where a potential drug offers a major advance in treatment.

Kalydeco was discovered in a collaboration between Vertex and the Cystic Fibrosis Foundation, which provided substantial scientific, financial and clinical support throughout the development process.

Vertex’s application for approval of Kalydeco, submitted to the FDA in October 2011, included results from Phase 3 clinical trials of the drug in people ages 6 and older with the G551D mutation of CF. The results 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 the placebo.

Additional Resources

• Read the Vertex press release.
• Learn more about Kalydeco in these FAQs.