Efficacy

CAYSTON improved respiratory symptoms, such as coughing, wheezing, and sputum production as measured by CFQ-R* score

  • The CAYSTON Prescribing Information does not present quantitative data on changes in CFQ-R
  • The FDA determined the CFQ-R was not validated in accordance with its Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims4
  • CFQ-R score has not been shown to correlate directly with a change in FEV14
  • Improvement in respiratory symptoms was noted for CAYSTON-treated patients vs placebo on the last day of drug treatment (9.7 points; 95% CI; 4.3, 15.1; p<0.001)1
  • Statistically significant improvements were seen in adults and pediatric patients, but were substantially smaller in adults
  • A difference in respiratory symptoms between treatment groups was present 2 weeks after completion of treatment, although the difference was smaller (6.3 points; 95% CI: 1.2, 11.4; p=0.015)1

*Cystic Fibrosis Questionnaire-Revised.


AIR-CF1: FOREST PLOT

Respiratory Symptoms Assessed in All Patients By Disease Severity and Age1

Explore the CAYSTON data on respiratory symptoms across disease severities and age ranges.

The CAYSTON Prescribing Information does not present quantitative data on changes in CFQ-R

The FDA determined the CFQ-R was not validated in accordance with its Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims4

CFQ-R score has not been shown to correlate directly with a change in FEV14

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

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CAYSTON

Question 1/4:

Show Observed Data

How do you think patients with moderate lung disease taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)

25 +
- -15

SEE ANSWER

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

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CAYSTON

Question 1/4:

Show Observed Data

How do you think patients with moderate lung disease taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)

You answered . The observed results for CAYSTON are shown in blue below.

NEXT QUESTION

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

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CAYSTON

Question 2/4:

Show Observed Data

How do you think patients with severe lung disease taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)

25 +
- -15

SEE ANSWER

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

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CAYSTON

Question 2/4:

Show Observed Data

How do you think patients with severe lung disease taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)

You answered . The observed results for CAYSTON are shown in blue below.

NEXT QUESTION

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

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CAYSTON

Question 3/4:

Show Observed Data

How do you think patients younger than 18 years and taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)

25 +
- -15

SEE ANSWER

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

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CAYSTON

Question 3/4:

Show Observed Data

How do you think patients younger than 18 years and taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)

You answered . The observed results for CAYSTON are shown in blue below.

NEXT QUESTION

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

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CAYSTON

Question 4/4:

Show Observed Data

How do you think patients 18 years and older taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)

25 +
- -15

SEE ANSWER

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

Placebo

CAYSTON

Question 4/4:

Show Observed Data

How do you think patients 18 years and older taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)

You answered . The observed results for CAYSTON are shown in blue below.

END QUIZ

Mean Change from Baseline CFQ-R* Respiratory Scores (±SE) at Day 28

CAYSTON AIR-CF1: Lung Function

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CAYSTON

Figure is adapted from Retsch-Bogart et al 2009.1

Statistically significant improvements were seen in both adult and pediatric patients, but were substantially smaller in adult patients. Two weeks after completion of treatment, a difference in respiratory symptoms between treatment groups was still present, though the difference was smaller.

Disclaimer: These questions are from the CFQ-R version for patients 14 years and older; younger children used a different questionnaire or a parent questionnaire was used. Minimum clinically important difference was not established for the child/parent questionnaire.

*Inferences regarding the treatment effect and size of the treatment effect should not be made based on the Respiratory Symptoms Scale of the CFQ-R.

Moderate disease: FEV1 >50% to ≤75% predicted; measured at screening.

Severe disease: FEV1 ≥25% to ≤50% predicted; measured at screening.


AIR-CF1: LUNG FUNCTION

CAYSTON SIGNIFICANTLY IMPROVED FEV1 LUNG FUNCTION

  • Mean percent change in FEV1 (L) was a prespecified secondary endpoint in AIR-CF1.1 Click here to review the study design
  • CAYSTON demonstrated a significant improvement in FEV1 (L) lung function with a placebo-adjusted treatment effect of 10% (95% CI: 6%, 14%) on Day 28
  • Improvements in FEV1 were comparable between adult and pediatric patients1
CAYSTON AIR-CF1: Lung Function
Kevin, treated with CAYSTON

“When I'm on CAYSTON, I notice a positive difference in my coughing and see a boost in my lung function.”

–Kevin, age 43, TREATED WITH CAYSTON

Individual results may vary.


AIR-CF1: DISEASE SEVERITY

AT DAY 28, 10% PLACEBO-ADJUSTED TREATMENT EFFECT IN FEV1 (L) WAS SEEN IN PATIENTS REGARDLESS OF BASELINE LUNG FUNCTION5

CAYSTON AIR-CF1: Disease Severity
  • At Day 28, there was a treatment effect of 10.3% (95% CI: 3%, 18%; p=0.0061) in patients with FEV1 ≥25% to ≤50%
  • At Day 28, there was a treatment effect of 10.1% (95% CI: 5%, 15%; p<0.0001) in patients with FEV1 >50% to ≤75%
  • Two weeks after completion of drug treatment, the difference in FEV1 between CAYSTON and placebo groups continued but decreased to 6% (95% CI: 2%, 9%)

AIR-CF1: ADULT/PEDIATRIC

IMPROVEMENTS IN FEV1 WERE COMPARABLE ACROSS ADULT AND PEDIATRIC PATIENTS ON DAY 28 OF TREATMENT5

CAYSTON AIR-CF1: Adult/Pediatric lung function chart
  • At Day 28, there was a treatment effect of 10.5% (95% CI: 6.4%,14.7%; p<0.0001) in patients ≥18 years
  • At Day 28, there was a treatment effect of 10.4% (95% CI: -1.3%, 22.1%; p=0.0790) in patients <18 years
  • Two weeks after completion of drug treatment, the difference in FEV1 between CAYSTON and placebo groups continued but decreased to 6% (95% CI: 2%, 9%)
Learn About AIR-CF1 Study Safety Profile
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