KALYDECO® Results

Age 12 years and older

 

Study 1

Study 1 was a 48-week study in which 161 people age 12 and older with a G551D mutation took KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours, along with their other cystic fibrosis (CF) treatments. Results were measured at different time points, including 24 and 48 weeks. All people took all their other CF treatments except for hypertonic saline through the entire length of the study (48 weeks). The study evaluated lung function (FEV1). Other results studied were CF respiratory symptoms, pulmonary exacerbations, weight, and sweat chloride. The study also evaluated the safety of KALYDECO.

 

Mutation eligible to enroll and included in this study was:

G551D.

 

Study results

Lung function (FEV1)

Lung function increased

10.6 percentage points greater average improvement, compared to placebo through 24 weeks.

10.5 percentage points greater average improvement, compared to placebo through 48 weeks.

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. 

 

Other study results

CF respiratory symptoms

8.6 points on average, compared to placebo, from the start of the study through Week 48.

Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

Pulmonary exacerbations

Pulmonary exacerbations decreased

Nearly 7 out of 10 people who took KALYDECO did not have a pulmonary exacerbation, compared to around 4 out of 10 people who took placebo at Week 48.

Pulmonary exacerbations are defined as changes in certain symptoms requiring changes in the use of oral, IV, or inhaled antibiotics.

Weight

Weight increased

About 6 pounds (2.7 kg) on average, compared to placebo at Week 48.

Sweat chloride

After taking KALYDECO:

Sweat chloride decreased on average by -48 mmol/L compared to placebo at Week 48.

Sweat chloride is the amount of salt in your child’s sweat. A decrease in sweat chloride levels does not mean there will be an improvement in lung function (FEV1). 

 

Study 7

Study 7 was an 8-week study in which people age 12 and older with 1 copy of the F508del mutation and a second mutation predicted to respond to KALYDECO took either KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours. All people took all their other CF treatments except for hypertonic saline. The study evaluated lung function (FEV1). CF respiratory symptoms and safety were also studied.

In this study, 156 people took KALYDECO (150-mg tablets) and 161 people took placebo.

 

Mutations enrolled in this study were:

2789+5G→A, 3272-26A→G, 3849+10kbC→T, 711+3A→G, A455E, D1152H, D579G, E831X, L206W, P67L, R1070W, R117C, R347H, R352Q, S945L, S977F.

Important considerations
  • Results varied by mutation. Not all mutations showed the same level of benefit when taking KALYDECO
  • Some people experienced less improvement compared to the average for all the people in the study. Others experienced more improvement
  • This is not intended to represent the full study design or results
  • Talk to your healthcare provider for more information

 

Study results

Lung function (FEV1)

Lung function increased

In the overall population studied, lung function increased by 4.7 percentage points on average compared to placebo from the start of the study to the average of Week 4 and Week 8.

Results varied by mutation.

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. 

 

Other study results

CF respiratory symptoms

CF respiratory symptoms improved

In the overall population studied, CF respiratory symptoms improved by 9.7 points on average compared to placebo from the start of the study to the average of Week 4 and Week 8.

Results varied by mutation.

Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

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