Tags: cystic fibrosis | drug | combo | shows | promise

Cystic Fibrosis Drug Combo Shows Promise in Treating Disease

By    |   Tuesday, 24 Jun 2014 06:17 PM

A new option for treating cystic fibrosis has taken a step closer to reality with Vertex Pharmaceuticals announcing Tuesday that late-stage trials of a combination of drugs were successful in treating the disease.

The company will submit applications for approval of the cystic fibrosis treatment this year, The New York Times reported.

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Results show increased lung function and reduced pulmonary flare-ups among patients who took the trial. The drugs also may help patients gain weight.

The lung function of those who took the drug combination for 24 weeks rose by 2.2 to 3.6 percentage points, while the lung function of those on a placebo declined slightly, the New York Times reported. Flare-ups were reduced by 30 percent to 40 percent.

The drug combination includes an experimental drug, lumacaftor, in combination with Vertex’s approved drug Kalydeco, The Wall Street Journal reported.

The improvement in lung function may not be noticeable to patients, but could be significant over time.

"Instead of there being a yearly decline which leads to early death, you're actually improving lung function," David Orenstein, a cystic-fibrosis specialist at Children's Hospital of Pittsburgh and the University of Pittsburgh Medical Center who helped conduct the research, told the Wall Street Journal. "If I can cut down their need for hospitalizations, or courses of antibiotics, that would be a discernible benefit."

The cystic fibrosis treatment could cost $160,000 per patient per year, Forbes reported.

“This is my bias as a physician,” Vertex CEO Jeffrey Leiden said, according to Forbes. “For me every improvement in lung function when you’re losing lung function, every day you don’t spend in the hospital, and every pound that you gain is quite meaningful. When you really talk to patients, that’s what matters.”

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A new option for treating cystic fibrosis has taken a step closer to reality with Vertex Pharmaceuticals announcing Tuesday that late-stage trials of a combination of drugs were successful in treating the disease.
cystic fibrosis, drug, combo, shows, promise
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2014-17-24
Tuesday, 24 Jun 2014 06:17 PM
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