Vertex Pharmaceuticals Inc. said a trial of its cystic fibrosis pill showed improvements in lung function in 6- to 11-year-olds in a 24-week trial, and will support its application for regulatory approval this year.
The study, the third of three phases generally required for approval, found that the children had an improvement in lung function of 12.5 percent from the beginning of the trial, the Cambridge, Massachusetts-based drugmaker said in a statement today. The patients had an improvement of 17.4 percent compared with the placebo group.
Vertex’s medicine aims to repair the effects of a genetic mutation found in about 4 percent of cystic fibrosis patients. Cystic fibrosis is an inherited disorder that causes build-ups of sticky mucus in the airways, leading to lung damage and infections that can kill patients before they turn 40. Success in treating children with the disorder could limit lung damage before it accumulates, RBC Capital Markets LLC analyst Jason Kantor said in a research note today.
“The greatest impact on clinical outcome will likely come from drugs that are effective in children, before their lung function deteriorates significantly,” said Kantor, who is based in San Francisco and recommends buying Vertex shares. “From a commercial perspective, starting chronic therapy in children and continuing through adulthood increases the size of the market substantially.”
Today’s results are an interim readout from a 48-week study, the company said. Vertex is also in the earlier stages of developing another pill that may help treat a majority of cystic fibrosis patients.
Vertex rose $1.56, or 3.3 percent, to $48.90 at 4 p.m. New York time in Nasdaq Stock Market composite trading. The shares have gained 19 percent in the last 12 months.
The company plans to seek U.S. and European clearance for VX-770 in the second half of this year. Kantor said the medicine may be on the market in the first half of 2012.
In a study released last month, Vertex reported that the medicine increased breathing function in adults by more than 10 percent in a year, reduced patients’ need for antibiotics and led to an average weight gain of 7 pounds. Children with cystic fibrosis have a hard time gaining weight. In this study, the children on the medicine gained an average of 8.1 pounds.