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Vertex cystic fibrosis combination succeeds in late-stage trials

collected by :Molly Tony

That is important as bronchial constriction has led up to 30 percent of those prescribed Vertex’s Orkambi combination treatment to stop taking it.
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.
Patient discontinuations of the Kalydeco/tezacaftor combination due to adverse side effects, including respiratory-related side effects, were low and similar to placebo.
By March 28 (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.
Vertex plans to apply for U.S. and European approvals in the third quarter.

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Vertex stock soars on promising data for cystic fibrosis drug

If OK’d, the combination drug would be Vertex’s third cystic fibrosis treatment to make it to the US market in the past six years.
Advertisement“A lot of Orkambi patients may be willing to move over to” the combination drug once it’s approved, Ramsey said.
Shares of Vertex Pharmaceuticals Inc. climbed more than 20 percent Wednesday, the day after the Boston biotech reported positive clinical data on an experimental treatment for the lung disease cystic fibrosis.
The findings — which showed that the two-drug combination improved patients’ lung function — signaled to investors that it stands a good chance of winning approval from regulators.
But she said one key measure, absolute improvement in lung function, was greater in the combination drug trial than in the Orkambi trial.

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