A journal article has recently been published about Kalydeco: http://err.ersjournals.com/content/22/127/66.full.pdf+html

Most of the information is not new, but there are a few interesting points:

“Nearly 75% of ivacaftor-treated adults had a mean improvement of 5% points (absolute). Improvements in patients with poor pulmonary function were similar to those in patients with only mild functional impairment. The responses in younger CF patients with mild disease were particularly striking, even for those with normal FEV1 %.”

“Preliminary results from PERSIST also strengthened evidence that ivacaftor has a beneficial effect on CF patients with severe disease. In this subgroup, the mean FEV1% on day 1 of STRIVE was 34.5. This improved to a mean absolute change of 10.8 at day 15 and a mean absolute change of 13.0 at week 12.”

“The F508del mutation not only has trafficking defects but also affects the function of the protein. The small amount of F508del CFTR protein that reaches the cell surface opens less frequently and does not stay at the cell membrane for as long as normal CFTR. Such a defect might, therefore, be accessible to potentiators, which improve the functional activity of the residual protein. In vitro studies have shown that ivacaftor also acts as a ‘‘modest’’ potentiator to the small amount of F508del CFTR channels that are trafficked to the cell surface.”


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1. Class 2 Mutations & F508del, 3. Vertex - Kalydeco