Vertex' ($VRTX) orphan drug Kalydeco boasts a comparatively small patient pool that the drugmaker is trying to swell, with an eye on adding cystic fibrosis patients ...

- KALYDECO® (ivacaftor) is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function ...

Please provide your email address to receive an email when new articles are posted on . Vertex Pharmaceuticals has received FDA approval for ivacaftor to treat cystic fibrosis among patients, aged 6 ...

Despite falling short of its primary endpoint in a trial to examine its effects in cystic fibrosis patients with the R117H mutation, Vertex's ($VRTX) Kalydeco has ...

Vertex Pharma filed the sNDA based on phase III study results. We note that although Kalydeco had not met the primary endpoint of mean absolute change from baseline compared to placebo in a phase III ...

Vertex Pharmaceuticals Inc. said that its cystic-fibrosis drug Kalydeco failed to show a statistically significant benefit during a late-stage study, casting uncertainty over whether the treatment ...

-Mean absolute treatment difference in lung function across all patients was 2.1 percentage points (p=0.20) and mean relative treatment difference was 5.0% (p=0.06)(n=69); study did not meet its ...

A Food and Drug Administration advisory committee has recommended approval of Vertex Pharmaceuticals Inc.’s cystic fibrosis drug to patients age 6 and older with the R117H mutation. The FDA’s ...

Vertex Pharma is looking to get Kalydeco approved for the treatment of patients (six years of age and above) with CF who have the R117H mutation in the cystic fibrosis transmembrane regulatory (:CFTR) ...

- If approved, KALYDECO® (ivacaftor) will be the first and only medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual ...