BMI Improvements Sustained for up to 120 Weeks1
Increases in mean BMI were seen in both ORKAMBI treatment groups1
Absolute change from baseline at Week 24 in BMI was a secondary endpoint in Trials 1 and 2 and was statistically significant in Trial 2, but not in Trial 1.2
Adapted from Konstan MW et al. Lancet Respir Med. 2017;5(2):107-118, with permission from Elsevier.
|a||In the individual analyses of these trials, changes were statistically significant with ORKAMBI vs placebo in Trial 2 (P<0.0001), but not statistically significant in Trial 1.2|
|b||Results are based on a pooled analysis that was not prespecified and did not correct for multiple comparisons.|
|c||Includes data from Trials 1 and 2.|
RATE OF CHANGE ANALYSIS
Improved BMI Rate of Change vs Matched Controls3
Analysis suggests ORKAMBI has a long-term impact on BMI
Adapted from Konstan MW et al. Lancet Respir Med. 2017;5(2) (suppl 1-28):107-118, with permission from Elsevier.
|a||Post-baseline data limited to 2 years. Visits ≤21 days of initiation excluded.3|
|b||Day 21 of Trials 1 and 2.1|
Trials 1 & 2 Study Design: Trials 1 and 2 were 24-week, Phase 3, randomized, double-blind, placebo-controlled trials of patients with CF who were homozygous for the F508del mutation and age 12 years and older. Patients received either ORKAMBI (lumacaftor 400 mg/ivacaftor 250 mg q12h) or placebo and continued to take their prescribed CF therapies.2
Extension Study Design: Patients who completed Trials 1 and 2 were eligible to enroll in a 96-week Extension Study. Of patients from Trials 1 and 2, 340 patients continued to receive ORKAMBI during the Extension Study and 176 rolled over from placebo to ORKAMBI.1
Rate of Change Analysis Design: The Rate of Change Analysis utilized data from 455 patients who received ORKAMBI in Trials 1 and 2, and the Extension Study. A matched cohort of 1588 patients from the US CFFPR was used as the comparator because there was no placebo group in the Extension Study. A propensity-score approach was used to match the two groups based on identified risk factors for lung function decline.1