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People with CF pictured may 
or may not be taking ORKAMBI.

ORKAMBI® Results

Keep in mind, results shown are an average for all people studied and differed among individuals. You or your child may have a different experience.


Study in Children 
Age 1 Through 2 Years

View the study details and results

Studies in Children 
Age 2 Through 5 Years

View the study details and results

Studies in Children 
Age 6 Through 11 Years

 

View the study details and results

Studies in People 
Age 12 Years and Older

 

View the study details and results

Safety study

The primary purpose of the 24-week safety study of 46 children age 1 through 2 years with 2 copies of the F508del mutation in their CFTR gene was to determine the safety of ORKAMBI.


The study also evaluated changes in sweat chloride levels.

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After 24 weeks, there was a 2-week period when ORKAMBI was stopped in order to observe any changes in sweat chloride. For the last 2 weeks of the study, children only took their other prescribed CF therapies and did not take ORKAMBI.

 

46 children took ORKAMBI

No children in the study took placebo.

How ORKAMBI was given

15 lbs to <20 lbs

Children who weighed 15 lbs to less than 20 lbs (7 kg to less than 9 kg) received lumacaftor 75 mg/ivacaftor 94 mg every 12 hours for 24 weeks.

20 lbs to <31 lbs

Children who weighed 20 lbs to less than 31 lbs (9 kg to less than 14 kg) received lumacaftor 100 mg/ivacaftor 125 mg every 12 hours for 24 weeks.

≥31 lbs

Children who weighed 31 lbs or more (14 kg or more) received lumacaftor 150 mg/ivacaftor 188 mg every 12 hours for 24 weeks.

All children:

  • Took 1 packet of ORKAMBI oral granules mixed with 1 teaspoon of soft food or liquid every 12 hours
  • Ate fat-containing food just before or just after taking their ORKAMBI dose
  • Continued to take their other prescribed CF therapies throughout the study

Study considerations

  • This study took place during the COVID-19 pandemic. As a result, Vertex put in place certain processes and guidelines during the study. Talk to your healthcare provider if you have any questions


Talk to your healthcare provider to learn more about how ORKAMBI was approved for children age 1 through 2 years.

Study results

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Safety

The safety of ORKAMBI, observed in the safety study, was similar to what was observed in people age 2 years and older.

See the possible side effects of ORKAMBI.

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Sweat Chloride

After taking ORKAMBI for 24 weeks:

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Sweat chloride decreased 29.1 mmol/L on average
(average mmol/L at beginning of study was 104.2)

After ORKAMBI was stopped for 2 weeks

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Sweat chloride increased 27.3 mmol/L on average

Sweat chloride is a measure of the amount of salt in a person’s sweat (mmol/L). Sweat chloride level measurement is used to help diagnose CF. High sweat chloride levels are a hallmark of CF. Because no one took placebo in the safety study, it is not known if changes in sweat chloride levels were due to ORKAMBI.

Changes in sweat chloride levels are not related to changes in lung function.

Safety studies

The safety of ORKAMBI in children age 2 through 5 years with 2 copies of the F508del mutation was studied for up to 2 years in a short-term (24-week) study and a long-term (96-week) study.

Both studies looked at safety. In addition, the studies measured sweat chloride and body mass index (BMI).

Sweat chloride levels are a measure of the amount of salt in a child’s sweat (mmol/L). This measurement is used to help diagnose CF. High sweat chloride levels are a hallmark of CF. After 24 weeks, there was a 2-week period when the children stopped ORKAMBI in order to observe any changes in sweat chloride.

Body mass index measures someone's weight relative to their height.

Studied for up to 2 years

Short-term study

24 weeks

60

CHILDREN TOOK

ORKAMBI

After a 2-week period in which participants did not take ORKAMBI, 57 children from the short-term study took part in the long-term study.

Long-term study

Additional 96 weeks (2 years)

57

CHILDREN TOOK

ORKAMBI

No children in the short-term and long-term studies took placebo.

How ORKAMBI was given

<31 lbs

Children who weighed less than ~31 pounds (less than 14 kg) received ORKAMBI oral granules (lumacaftor 100 mg/ivacaftor 125 mg) every 12 hours.

≥31 lbs

Children who weighed ~31 pounds or more (14 kg or more) received ORKAMBI oral granules (lumacaftor 150 mg/ivacaftor 188 mg) every 12 hours.

Children who turned 6 years old during the long-term study were switched from oral granules to tablets (lumacaftor 200 mg/ivacaftor 250 mg) every 12 hours.

All children:

  • Took 1 packet of ORKAMBI oral granules mixed with 1 teaspoon of soft food or liquid every 12 hours
  • Ate fat-containing food just before or just after taking their ORKAMBI dose
  • Continued to take their other prescribed CF treatments throughout the short-term and long-term studies. No children in the study took placebo

Study considerations

  • Because no one took placebo, it is not known if changes in sweat chloride levels and BMI were due to ORKAMBI
  • Changes in sweat chloride levels are not related to changes in lung function
  • ORKAMBI was approved for children age 2 through 5 years old based on the benefits shown in studies of ORKAMBI in people age 12 years and older, as well as the safety assessment of the short-term (24-week) study
Test

Safety

SHORT-TERM STUDY
The safety seen in both the short-term study and long-term study was similar to what was observed in children age 6 years and older.
Test

Sweat
Chloride

After taking ORKAMBI for 24 weeks

Decreased -31.7 mmol/L on average
(average mmol/L at the beginning of the study was 105.8)

 

AFTER ORKAMBI WAS STOPPED FOR 2 WEEKS:
Increased 33.0 mmol/L on average

Test

BMI

Increased 0.27 kg/m2 on average

Test

Safety

LONG-TERM STUDY

The safety seen in both the short-term study and long-term study was similar to what was observed in children age 6 years and older.

See the possible side effects of ORKAMBI

Test

Sweat Chloride

After taking ORKAMBI for 96 weeks

Decreases in sweat chloride were generally maintained

Test

BMI

Increases in BMI were generally maintained

Additional safety information

In the short-term study, ORKAMBI was discontinued for 3 children because of high liver enzymes in the blood. In the long-term study, ORKAMBI was discontinued for 2 children because of this side effect.

These are the most common side effects seen in the studies of children age 2 through 5 years. Note that no one in these studies took placebo.

 

SIDE EFFECTS SHORT-TERM STUDY
(60 CHILDREN)
Cough 63%
Vomiting 28%
Fever 28%
Runny nose 25%
Nasal congestion not reported

 

SIDE EFFECTS LONG-TERM STUDY
(57 CHILDREN)
Cough 83%
Vomiting 30%
Fever 40%
Runny nose 32%
Nasal congestion 44%

 

 

See below for results from a safety study and an efficacy and safety study.

Safety study

The primary purpose of the 24-week safety study of 58 children age 6 through 11 years with 2 copies of the F508del mutation in their CFTR gene was to determine the safety of ORKAMBI.

The study also evaluated changes in lung function and sweat chloride levels.

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Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second. Lung function was part of the safety assessment in this trial.

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Sweat chloride is a measure of the amount of salt in a person’s sweat (mmol/L). Sweat chloride level measurement is used to help diagnose CF. High sweat chloride levels are a hallmark of CF.

After 24 weeks, there was a 2-week period when ORKAMBI was stopped in order to observe any changes in results. For the last 2 weeks of the study children only took their other prescribed CF therapies and did not take ORKAMBI.

How ORKAMBI was given

All children in the study took 2 tablets of ORKAMBI (each containing lumacaftor 100 mg/ivacaftor 125 mg) every 12 hours with fat-containing food. No children in the study took placebo. All children continued to take their other prescribed CF treatments throughout the full study, including the 2-week period when they stopped taking ORKAMBI.

Safety study results

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Safety

The safety of ORKAMBI, observed in the safety study, was similar to what was observed in people age 12 years and older. Additional side effects in patients age 6 through 11 were observed in another study shown below.
See the possible side effects of ORKAMBI

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Lung Function (FEV1

upAfter taking ORKAMBI for 24 weeks:
FEV1 increased 2.5 percentage points

 

downAfter ORKAMBI was stopped for 2 weeks:
FEV1 decreased 3.2 percentage points

 

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Sweat Chloride

arrow-purple-down-copy@2x.pngAfter taking ORKAMBI for 24 weeks:
Sweat chloride decreased 24.8 mmol/L on average
(average mmol/L at beginning of study was 105.9)

 

arrow-purple-up-copy@2x.pngAfter ORKAMBI was stopped for 2 weeks:
Sweat chloride increased 21.3 mmol/L on average

 

Because no one took placebo in the safety study, it is not known if changes in lung function and sweat chloride levels were due to ORKAMBI.

Safety study considerations

  • Changes in sweat chloride levels observed in the safety study are not related to changes in lung function

Efficacy and safety study

The primary purpose of the 24-week efficacy and safety study of 204 children with 2 copies of the F508del mutation in their CFTR gene was to determine the efficacy of ORKAMBI on lung function based on lung clearance index (LCI). LCI is a measure of lung function that determines how well the lungs are working. This study also evaluated the safety of ORKAMBI.

How ORKAMBI was given

In this study, 103 children took 2 tablets of ORKAMBI (lumacaftor 100 mg/ivacaftor 125 mg) every 12 hours with fat-containing food. The remaining 101 children took placebo every 12 hours with fat-containing food. All children continued to take their other prescribed CF therapies. 

Efficacy and safety study results

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Lung Clearance Index (LCI)

arrow-purple-up-copy@2x.pngAfter taking ORKAMBI for 24 weeks:
Lung function was improved versus placebo, based on LCI

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Safety

The safety of ORKAMBI, observed in the efficacy and safety study, was similar to what was observed in people age 12 years and older, with the addition of these side effects: cough with sputum, stuffy nose, headache, stomach pain, and increase in sputum.
See the possible side effects of ORKAMBI

Study considerations

  • The efficacy results from the efficacy and safety study are not included in the full Prescribing Information. Also, the FDA did not consider the results of this study when approving ORKAMBI

Study results suggest that ORKAMBI could 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.

How ORKAMBI was studied

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

24 weeks

2 Short-Term Studies 

  • In the short-term studies, ORKAMBI and placebo were compared. 369 people took ORKAMBI and 371 people took placebo
  • 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 therapies

After 24 weeks, people from the short-term studies were able to participate in the long-term study.

96 weeks

1 Long-Term Study 

  • In the long-term study, all participants took ORKAMBI
  • At the start of the long-term study, there were 2 groups: 340 people who continued taking ORKAMBI, and 176 people who switched to ORKAMBI from placebo
  • Every 12 hours, participants took 2 tablets of ORKAMBI (lumacaftor 200 mg/ ivacaftor 125 mg) with fat-containing food. People continued to take their other prescribed CF therapies
  • The main focus of the long-term study was to evaluate the safety of treatment with ORKAMBI

Long-term study 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

Study results

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Lung Function (FEV1

Lung function improved in both short-term studies (24 weeks) compared  to placebo

arrow-purple-up-copy@2x_1_0.png Study 1 FEV1 increased by 2.6 percentage points

arrow-purple-up-copy@2x_1_0.png Study 2 FEV1 increased by 3.0 percentage points

 

Lung function maintained above where it was before treatment in the 
long-term study (96 weeks)


No one in the long-term study took placebo for comparison. Therefore, it cannot be determined if the changes were due to ORKAMBI.

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Body Mass Index (BMI)

BMI increased in both short-term studies (24 weeks) compared to placebo

arrow-purple-up-copy@2x_1.pngStudy 1 BMI increased by 0.1 kg/m2
For example: +0.7 pounds for a person who is 5’ 4” and weighs 
110 lbs

arrow-purple-up-copy@2x_1.pngStudy 2 BMI increased by 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 in the long-term study (96 weeks)

No one in the long-term study took placebo for comparison. Therefore, it cannot be determined if the changes were due to ORKAMBI.

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CF Respiratory Symptoms

CF respiratory symptoms in both short-term studies (24 weeks) were similar to placebo

arrow-purple-up-copy@2x_1_4.png Study 1 CF respiratory symptoms increased by 1.5 points

arrow-purple-up-copy@2x_1_4.png Study 2 CF respiratory symptoms increased by 2.9 points

 

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

 

CF respiratory symptoms were measured over time in the long-term study (96 weeks). No one in the long-term study took placebo for comparison.


About CF Respiratory Symptoms:

  • Includes cough, difficulty breathing, and amount of mucus coughed up
  • Respiratory symptoms were measured using a tool called the Cystic Fibrosis Questionnaire-Revised Respiratory Domain score
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Pulmonary Exacerbations

Pulmonary exacerbations were less likely in both short-term studies (24 weeks) compared to placebo

arrow-purple-up-copy@2x_1.pngStudy 1 pulmonary exacerbations decreased by ~30%

arrow-purple-up-copy@2x_1.pngStudy 2 pulmonary exacerbations decreased by ~40%

 

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

 

A reduction in the number of pulmonary exacerbations per year was maintained over time during the long-term study (96 weeks).


No one in the long-term study took placebo for comparison. Therefore, it cannot be determined if the changes were due to ORKAMBI.


From a separate analysis combining the results of the 2 short-term studies

arrow-purple-up-copy@2x_1.png56% less likely to have a pulmonary exacerbation that requires intravenous antibiotics

arrow-purple-up-copy@2x_1.png61% 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.

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Safety

The safety profile for people age 12 years and older, including Serious and Common Side Effects, was established through these 2 short-term (24 weeks) studies. 

Safety in the long-term (96 weeks) study was generally consistent with what was seen in the 2 short-term studies.

No one in the long-term study took placebo for comparison.

Measuring the results of treatment

After beginning treatment, you or your child may not always feel a difference on a day-to-day or even week-to-week basis. That’s why it’s important to know what to look for and to discuss your results with your doctor. You may want to review certain measures with your doctor to compare them with how they were when treatment was first started.