CF News

There are some exciting new advancements in pharmeceutical development for CF and it is so encouraging to read about this research coming down the pipeline in the search for a cure. We will continue to pray that, one day, something will be found to treat and cure Kelsie's form of CF. By the way, she has the most common form of CF with 2 copies of the delta F508 mutation (the one mentioned at the end of the 2nd article.)


Wall Street Journal: “At Last, Cystic Fibrosis Drugs Seem Near”

October 20, 2011

Drug makers are edging closer to new treatments for cystic fibrosis that could stop the destructive impact of the disease, says an article in The Wall Street Journal today.

"We really believe with these new drugs that we should be able to add decades to the lives of patients in a very short period of time," Robert J. Beall, Ph.D., president and chief executive of the Cystic Fibrosis Foundation, says in the article.

The drug that’s furthest along is a pill from Vertex Pharmaceuticals, which on Wednesday filed for regulatory approval of its drug Kalydeco (VX-770). If approved, it will be the first drug on the market that targets the underlying cause of CF.

The CF Foundation played a significant role in the development of the potential drug, which is for people age 6 and older who carry at least one copy of the G551D mutation of cystic fibrosis.

The story also highlights 8-year-old Capri Faulk, who has CF and participated in the Kalydeco clinical trials. Since starting on the medicine in 2009, she has added 24 pounds to her 49-pound frame, and can play outside with her brother and sister without getting exhausted, her mother Nicole Faulk says in the article.

"I had hopes and dreams for her, and I was scared they would be robbed from her and from me" until the new drug came along, she said.
Link to Wall Street Journal article




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.

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.

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.

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.

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.