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Vertex Pharmaceuticals (MA) (VRTX) Trial Suggests Kalydeco May Work In More Cystic Fibrosis Patients



6/5/2014 9:46:50 AM

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It’s not the trial results that most investors are waiting for out of Vertex Pharmaceuticals, but the results of a mid-stage trial of the company’s approved cystic fibrosis drug point toward a possible doubling of the patients in which the drug works in a few years. Today the Boston-based biotech announced good results from a small, mid-stage study of its drug, Kalydeco, in 24 patients with cystic fibrosis due to residual function mutation. That cause of the inherited disease — which makes it difficult to breathe and is life-threatening — is shared by about 3,000 people ages 6 and older in North America, Europe and Australia — areas where the number of patients can be accurately determined, according to the company. By contrast, there are 2,400 cystic fibrosis patients worldwide with one of the nine specific genetic mutations for which Kalydeco is already approved.

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