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Vertex Pharmaceuticals Incorporated Message Board

  • qdelfan qdelfan Jun 4, 2014 8:30 AM Flag

    Positive proof of concept study just announced using kalydeco monotherapy on residual function mutations

    Phase 3 study to be started which would allow 3000 more patients to be approved to use kalydeco monotherapy.

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    • 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.

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