New Vertex drugs show dramatic gains for tough-to-treat cystic fibrosis patients

Cystic fibrosis patients with genetic mutations that resist other treatments gained in lung function in trials of three new Vertex combination therapies.

Vertex Pharmaceuticals scored a major win Tuesday with the release of data from three clinical trials testing three different triple combinations of cystic fibrosis drugs. Patients genetically resistant to all treatments now on the market showed unprecedented gains in lung function on all three experimental therapies.

The Vertex data point everyone will be gawking at: a 10 percentage point improvement, adjusted for placebo, in FEV1, an important measure of lung capacity. This significant gain was reported for all three triple-drug regimens being tested. The patients enrolled in the three studies have cystic fibrosis caused by a genetic mutation known as F508del/Min,

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