vertex cystic fibrosis

Vertex Pharmaceuticals Offers Promising Cystic Fibrosis Drug

Promising New Cystic Fibrosis Drug

Reuters New Service has reported that Vertex Pharmaceuticals Inc said on Tuesday its Kalydeco cystic fibrosis treatment, when given with an experimental drug, demonstrated significant improvements in lung function in Cystic Fibrosis patients.  This occurred in two 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. This is important in that bronchial constriction has led up to 30 percent of those prescribed Vertex’s Orkambi combination treatment to stop taking it. Anytime there is a serious adverse reaction to a drug, the user should speak with their physician about discontinuation of the drug.

“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 of 2017.   Business Insider

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