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Vertex Pharmaceuticals Incorporated
today announced data from a
Phase 3 study of ivacaftor in 69 people 6 years of age and older with cystic
fibrosis (CF) who have the R117H mutation. In the study, the mean absolute
treatment difference in the change from baseline in percent predicted
FEV[1] between treatment with ivacaftor and placebo was 2.1 percentage points
(p=0.20) and the mean relative treatment difference in percent predicted
FEV[1] was 5.0 percent (p = 0.06) through the 24-week treatment period among
all patients (intent-to-treat analysis). The study did not meet its primary
endpoint of the absolute change from baseline in FEV[1] (percent predicted
forced expiratory volume in one second, FEV[1]) throughout the treatment
period for ivacaftor compared to placebo across all patients. A pre-specified
subset analysis in patients 18 years of age and older (n=50) showed
statistically significant improvements in lung function and other key
secondary endpoints. In these patients, the mean absolute treatment difference
in percent predicted FEV[1] between treatment with ivacaftor and placebo was
5.0 percentage points (p=0.01) and the mean relative treatment difference in
percent predicted FEV[1] was 9.1 percent (p=0.008) through the 24-week
treatment period. Vertex believes that the results show a clinical benefit for
patients age 18 and older with the R117H mutation. The company plans to meet
with the U.S. Food and Drug Administration (FDA) in early 2014 to discuss
these data and the potential submission of a supplemental New Drug Application
(sNDA) for people with the R117H mutation.
R117H is the most common residual function mutation. In North America, Europe
and Australia, approximately 1,100 people with CF ages 6 and older have at
least one copy of an R117H mutation. In the United States, approximately 300
people have the R117H mutation and are 18 years of age or older.
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