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Vertex Cystic Fibrosis Drug Combo Improves Lung Function in Study

June 24, 2014

Vertex Pharmaceuticals said Tuesday that a combination of its drugs lumacaftor and ivacaftor significantly improved lung function in patients with cystic fibrosis, and that the company plans to submit marketing applications in multiple countries.

After the 24-week study of 1,100 people with CF ages 12 and older, average lung function improved from 4.3 percent to 6.7 percent compared with placebo. The combo also reduced pulmonary flare-ups, which can lead to hospitalization, the Boston-based company said.

Lung function was assessed by the maximum a person could exhale in one second, the company said. At the start of the trial, the patients had average lung function 61 percent of what would be predicted for people their age.

The drugmaker said the combination was well tolerated, with the most common adverse events being cough, headache, infective pulmonary exacerbation and increased sputum.

The drugmaker’s Ivacaftor CF drug was approved in 2012, but only works against mutation found in about 4 percent of patients. The addition of the experimental lumacaftor, would make the combo useable for nearly half of CF patients, those with two copies of the most common F508del mutation, the company said.

Vertex said it would submit marketing applications to the FDA and in Europe in the fourth quarter of 2014.

Cystic fibrosis leads to infections and lung damage due to mucus build up in the lungs. About 30,000 Americans and 75,000 globally have the disease, which causes many to die before 40. — Kellen Owings

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