Clinical Trial Results

How ORKAMBI® was studied in people age 12 years and older

Two 24-week clinical trials were done to evaluate the safety and efficacy of ORKAMBI in people age 12 years and older with 2 copies of the F508del-CFTR mutation.

In Trial 1, 182 people took ORKAMBI every 12 hours with fat-containing food, and 184 people took placebo (sugar pill) every 12 hours with fat-containing food.

In Trial 2, 187 people took ORKAMBI every 12 hours with fat-containing food, and 187 people took placebo (sugar pill) every 12 hours with fat-containing food.

In both trials, the ORKAMBI group and the placebo group continued to take all of their other prescribed cystic fibrosis (CF) therapies.

Talk to your healthcare provider about these trial results and if ORKAMBI is right for you.

Benefits
of
Orkambi

For people age 12 years and older

People Taking Orkambi Had Improved And Sustained Lung Function Through 24 WEEKS.

IMPROVEMENT IN BOTH TRIALS WAS MEASURED BY LOOKING AT THE AVERAGE CHANGE IN LUNG FUNCTION AT WEEKS 16 AND 24. LUNG FUNCTION WAS MEASURED IN A TEST CALLED FEV1, OR FORCED EXPIRATORY VOLUME IN 1 SECOND, WHICH IS DEPICTED IN THE IMAGES BELOW.

Compared With Placebo, People Taking Orkambi HAD, ON AVERAGE:


Additional
Trial Results

During These Two 24-week Trials, The following Were Also Studied.

Compared With Placebo, People Taking Orkambi HAD, ON AVERAGE:

Compared with placebo, people taking ORKAMBI had, on average:

Compared with placebo, people taking ORKAMBI had, on average:



How ORKAMBI® was studied in children age 6 through 11 years

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A 24-week trial was done to evaluate the safety of ORKAMBI in children age 6 through 11 with 2 copies of the F508del-CFTR mutation.

There were 58 people in this trial, and they all took 2 tablets of ORKAMBI every 12 hours with fat-containing food, along with their other prescribed cystic fibrosis (CF) therapies. None were given placebo.

After 24 weeks, treatment with ORKAMBI was stopped, and children continued in the study for an additional 2 weeks, taking only their other prescribed CF therapies.

The primary purpose of this study was to evaluate the safety of treatment with ORKAMBI in children age 6 through 11 with 2 copies of the F508del-CFTR mutation.

Safety was assessed by a variety of measures, including looking at side effects and evaluating changes in lung function. Another purpose of this study was to look at changes in sweat chloride.

Clinical Trial Results

Safety profile

The side effects from this study were similar to what was observed in trials of ORKAMBI in older patients. Review the possible side effects of ORKAMBI.

Additional Trial Results

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Lung function

Lung function was part of the safety assessment in this trial. Lung function was measured in a test called FEV1, or forced expiratory volume in 1 second.

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From the start of therapy to Week 24, children taking ORKAMBI and their other prescribed CF therapies had, on average, an increase in lung function of 2.5 percentage points.

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At Week 26, after 2 weeks of stopping treatment with ORKAMBI and continuing with only their other prescribed CF therapies, children experienced, on average, a decline in lung function of 3.2 percentage points.

Because no one took placebo, no comparisons could be made. Also, it is not known if changes in FEV1 were due to ORKAMBI.

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Sweat chloride

Sweat chloride is a measure of the amount of salt in your child’s sweat. People with CF have high levels of sweat chloride.

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From the start of therapy to Week 24, children taking ORKAMBI and their other prescribed CF therapies had, on average, a reduction in sweat chloride of 24.8 mmol/L.

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At Week 26, after 2 weeks of stopping treatment with ORKAMBI and continuing with only their other prescribed CF therapies, an increase in sweat chloride was observed of 21.3 mmol/L.

Because no one took placebo, no comparisons could be made. Changes in sweat chloride levels are not related to changes in lung function. It is not known if changes in sweat chloride levels were due to ORKAMBI.