Phase 3 clinical trials of ivacaftor (Kalydeco™) in combination with lumacaftor (VX-809) for people with two copies of the F508del mutation of cystic fibrosis showed significant improvements in lung function and other key measures of the disease, according to Vertex Pharmaceuticals Inc.This is very good news for my family as Baby Madeline has this mutation. It will be sometime before she can get the treatment. But as Cystic Fibrosis is a progressive disease in children doing more damage as the children age, I have more hope for her health than I did yesterday.
Based on these results, Vertex plans to submit a New Drug Application (NDA) by the end of 2014 to the U.S. Food and Drug Administration (FDA) for review, with possible approval in 2015.
Results from the two 24-week clinical trials mark an important milestone for nearly 50 percent of people with cystic fibrosis — specifically those with two copies of F508del, the most common CF mutation. The potential treatment is the first to combine two pills to address the underlying genetic cause of CF in people with the F508del mutation.
“This is a proud day for the CF community,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Many people with CF and their families have been eagerly awaiting these results, and we are thrilled with the outcome. These studies further validate that we are on the right track to getting new and effective treatment into the hands of people with CF who so desperately need them.”
The trials tested two different doses of the ivacaftor and lumacaftor combination therapy. Compared with those on placebo, participants who took the combination treatment showed significant and consistent improvement in lung function and in other important health measures, including weight gain, and a reduction in the rate of pulmonary exacerbations.
8:20 AM PT: Here is the wiki for Cystic Fibrosis.
It is a very real nightmare and a true monster.