We just published our report on Vertex’s experimental drugs for CF: VX-770 is generating a lot of buzz among experts and patients. We examined what this excitement is all about and what it could mean for patients and – of course – for Vertex. In a nutshell, VX-770 is a CFTR modulator, a new treatment approach that attempts to fix the genetic defect that causes cystic fibrosis. If successful, it could theoretically alter the natural history of the disease. This would be huge for patients. The current phase III trial is focusing on patients with the G551D mutation, which is not very common. The million (or billion) dollar question for Vertex is not so much related to VX-770’s effects in the G551D mutation, but rather whether it might work on the ubiquitous F508del mutation. This mutation affects up to 90% of the patients. Our report details the history of VX-770 and its prospects for success in phase III and beyond … a fascinating story worth following closely.


