This is great news. A few points to help understand significance. The residual function trial has a spectrum of different mutations so the benefit for some will be much greater than others, so the 4.6 ish relative and 7.5ish absolute improvement is very solid since some could be benefitting more and others less. This will offer opportunity to get a broader label and sweep in mutations which might have only gotten a 2-3 % benefit. Also, that fev improvement is great considering these are residual function and r117 residual function mutation only showed improvement for those over 18 meaning it is harder to show benefit, but this trial did. So anyone thinking this is minimal benefit compared to 551 on kalydeco don't underestimate the results...good enough for approval with a broad net of mutations and will bring help to CFers in a pill that is equivalent to pulmozyme but better because it helps all aspects of disease and not just lungs.
Verity, I agree this is great news. By the way, what happened regarding the FDA's decision to approve Kalydeco monotherapy to treat r117 residual function patients age 18 and older? I thought Vertex had been in discussions with the FDA over the past six months to apply for this expanded use of Kalydeco based on the data from the phase 3 trial announced back in December?