Vertex cystic fibrosis combination succeeds in late-stage trials

[March 29, 2017] By Bill Berkrot

(Reuters) - Vertex Pharmaceuticals Inc said on Tuesday its Kalydeco cystic fibrosis treatment given with an experimental drug demonstrated significant improvements in lung function in a pair of late-stage trials the company plans to use to seek approval of the combination therapy.

Patient discontinuations of the Kalydeco/tezacaftor combination due to adverse side effects, including respiratory-related side effects, were low and similar to placebo. That is important as bronchial constriction has led up to 30 percent of those prescribed Vertex's Orkambi combination treatment to stop taking it.

"If we could get the same efficacy but have a cleaner, safer drug (than Orkambi) that would be a win and the results are actually better than that," said Dr. Patrick Flume, an investigator on both trials.

"I think they should be very excited about the news," added Flume, professor of medicine and pediatrics at Medical University of South Carolina, who said he expects the combination to win approval.

Vertex plans to apply for U.S. and European approvals in the third quarter.

CF is a rare, life-shortening genetic disease affecting about 75,000 people in North America, Europe and Australia that causes progressive lung damage and loss of lung function. The studies tested CF patients aged 12 and over.

(Reporting by Bill Berkrot in New York; Editing by Diane Craft and Matthew Lewis)

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