Some doubts for Kalydeco in R117H patients

enniob

Administrator
Staff member
Vertex would like to add patients with R117H mutations into the Kalydeco patient pool. But they are facing some resistance from the FDA. R117H mutation "results in a functionally different chloride channel than with the G551D mutation", Kalydeco's original indication. In a trial to examine R117H, requested by the FDA, Kalydeco missed its primary endpoint. But Vertex has said it believes there's hope for the drug in that population, potentially in a subpopulation of patients, like those over 18 years old. The FDA "question whether there is enough sufficient evidence for approval." There is an estimated 1110 patients with the R117H mutation compared to 2700 with the G551D mutation which is the one best treated by Kalydeco.

Read source: http://t.co/Z1qGWLLgv3

- Chris
 
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