New England Journal of Medicine publishes Phase II study of VX-770 as a new approach to treat the underlying cause of cystic fibrosis.(Report)

In a study published in New England Journal of Medicine, treatment with a new drug candidate known as VX-770 resulted in improvements in lung function and markers of disease in a Phase II clinical trial of 39 people with cystic fibrosis (CF). There were no discontinuations due to adverse events in the study, and the frequency of adverse events was similar across the study groups. VX-770 is an oral (tablet) medicine that is being developed by Vertex Pharmaceuticals Inc to directly target the defective protein known to cause CF. An accompanying editorial on this study was also published in New England Journal of Medicine. Vertex Pharmaceuticals retains worldwide rights to develop and …