Measuring the Results With ORKAMBI®

Study Results Suggest That ORKAMBI Can Have an Impact for up to 2 Years

The possible benefits and risks of treatment with ORKAMBI in people with cystic fibrosis (CF) age 12 years and older with 2 copies of the F508del mutation were studied in 2 Short-Term Studies of 24 weeks. People who completed these studies were able to participate in a Long-Term Study for an additional 96 weeks.

Below, you'll find more details on how ORKAMBI was studied, as well as the study results. All of the results shown for each study below are an average of all people evaluated in each study.

Studied up to 2 Years 2 Short-Term Studies
1 Long-Term Study

The possible benefits and risks of treatment with ORKAMBI were studied for up to 2 years in people with CF age 12 and older.

  • In the Short-Term Studies, ORKAMBI and placebo (sugar pill) were compared. In the Long-Term Study, all participants took ORKAMBI
  • Every 12 hours, participants took 2 tablets of ORKAMBI (lumacaftor 200 mg/ivacaftor 125 mg) or placebo, with fat-containing food. People continued to take their other prescribed CF treatments

Considerations

  • The Long-Term Study did not have any participants who took placebo. All patients knew they were taking ORKAMBI, which may have influenced the results
  • The FDA did not consider the Long-Term Study when approving ORKAMBI, and it is not included in the full Prescribing Information. The Long-Term Study may not meet the FDA’s definition of an acceptable study because there was no placebo group included for comparison
  • At around Week 72, almost half the participants stopped taking part in the Long-Term Study because ORKAMBI was approved by the FDA at that time. Participants could start getting ORKAMBI from their own doctor.

Lung Function Improved in Both Studies

STUDY 1
2.6 Percentage Points
STUDY 2
3.0 Percentage Points

Lung Function Maintained Above
Where It Was Before Treatment

Because no one in the Long-Term Study took placebo for comparison, it cannot be determined if the changes were due to ORKAMBI.

BMI Increased in Both Studies

STUDY 1 0.1 kg/m2

For example:

+0.7 pounds for a person

who is 5'4" and weighs 110 lbs

STUDY 2 0.4 kg/m2

For example:

+2.4 pounds for a person

who is 5'4" and weighs 110 lbs

Based on statistical analysis, it cannot be determined if the changes in Study 1 were due to ORKAMBI.

BMI Increased Over Time

Because no one in the Long-Term Study took placebo for comparison, it cannot be determined if the changes were due to ORKAMBI.

CF Respiratory Symptoms in Both Studies
Were Similar to Placebo

STUDY 1 1.5 Points
STUDY 2 2.9 Points

Based on statistical analysis, it cannot be determined if these changes were due to ORKAMBI.

CF Respiratory Symptoms Were Measured Over Time

Because no one in the Long-Term Study took placebo for comparison, it cannot be determined if the changes were due to ORKAMBI.

Pulmonary Exacerbations Were Less Likely in Both Studies

STUDY 1 30%
STUDY 2 40%

Based on statistical analysis, it cannot be determined if these changes were due to ORKAMBI.

A Reduction in the Number of Pulmonary Exacerbations per Year Was Maintained Over Time

Because no one in the Long-Term Study took placebo for comparison, it cannot be determined if the changes were due to ORKAMBI.

From a separate analysis combining
the results of both studies

56% less likely to have a pulmonary exacerbation that requires intravenous antibiotics

61% less likely to have a pulmonary exacerbation that requires hospitalization

This analysis was not planned as part of the original study, nor is it included in the full Prescribing Information. It cannot be determined if the observed changes were due to ORKAMBI.

Change the Way Your Child’s CF Is Treated

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

The Study Evaluated:

LUNG FUNCTION

Lung function is measured with an FEV1 test, which measures how much air a person can exhale in a forced breath. Lung function was part of the safety assessment in this trial.

Sweat Chloride Levels

Children with CF have high levels of sweat chloride (mmol/L), which is a measure of the amount of salt in a child’s sweat. Children with sweat chloride levels of less than 30 mmol/L are unlikely to have CF.

The side effects from this study were similar to what was observed in studies of ORKAMBI in older people. See the side effects here >>>

After taking ORKAMBI for 24 weeks, lung function increased and sweat chloride levels decreased.

After ORKAMBI was stopped, changes were seen in lung function and sweat chloride levels.

Because patients did not take placebo, no comparisons could be made.

It is not known if changes in lung function and sweat chloride levels were due to ORKAMBI. Changes in sweat chloride levels are not related to changes in lung function.