A TEAE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship that occurred or worsened in the period extending from the first dose of study drug to 6 weeks after the last dose of study drug. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. AE severity was graded as follows: Grade 1: mild; Grade 2: moderate; Grade 3: severe; Grade 4: life-threatening; Grade 5: fatal. A TEAE was considered related if in the opinion of the Investigator it was possibly or probably caused by the study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the Adverse Events module.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug.

A TELA is any abnormal laboratory value that started or worsened after administration of study drug. Abnormal values were defined as values outside normal range. Values considered abnormal included -Hepatic: Serum total bilirubin ≥1.5*upper limit of normal (ULN); serum gamma glutamyl transferase >2.5*ULN; serum alanine aminotransferase increase of >150 units/liter (U/L) without increased creatine kinase; -Adrenal: plasma adrenocorticotropic hormone >ULN (normal cortisol); -Renal: serum cystatin C >1.33 milligrams (mg)/L; serum creatinine >ULN-1.5*ULN for age; serum blood urea nitrogen ≥1.5*ULN; urine protein:creatinine >0.40 mg/deciliter (dL):mg/dL; urine protein:osmolality >0.30 mg/L:milliosmoles/kilogram; urine blood 2+; - Serum Electrolytes: serum sodium >150 millimoles (mmol)/L, <130 mmol/L; serum potassium >5.5, <3.0 mmol/L; serum magnesium >1.23 mmol/L, <0.5 mmol/L; total serum calcium >2.9 mmol/L, <2.0 mmol/L; serum phosphorous <0.8 mmol/L; serum biocarbonate- <16 mmol/L.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was validated by using current guidelines of the American Thoracic Society (ATS) and European Respiratory Society (ERS). Baseline was defined as Week 1 or the most recent value of percent-predicted FEV1 prior to the first dose of open-label treatment in Study 009e.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was validated by using current guidelines of the American Thoracic Society (ATS) and European Respiratory Society (ERS). The percentage of change in percent-predicted of FEV1 was calculated as follows: ((percent-predicted FEV1-Baseline percent-predicted FEV1)/Baseline percent-predicted FEV1)*100. Baseline was defined as Week 1 or the most recent value of percent-predicted FEV1 prior to the first dose of open-label treatment in Study 009e. A positive change from Baseline indicates that FEV1 improved.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was validated by using current guidelines of the ATS and ERS. Analyses with the Completer populations were performed to complement the analyses of the Study 009e As-Treated population. Baseline was defined as Week 1 or the most recent value of percent-predicted FEV1 prior to the first dose of open-label treatment in Study 009e.

Study 009/009e 96-Week Completer Population: participants who completed 48 weeks of treatment with ataluren or placebo in Study 009 and at least 48 weeks of treatment with ataluren in Study 009e. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was validated by using current guidelines of the ATS and ERS. The percentage of change in percent-predicted of FEV1 was calculated as follows: ((percent-predicted FEV1-Baseline percent-predicted FEV1)/Baseline percent-predicted FEV1)*100. Analyses with the Completer populations were performed to complement the analyses of the Study 009e As-Treated population. Baseline was defined as Week 1 or the most recent value of percent-predicted FEV1 prior to the first dose of open-label treatment in Study 009e. A positive change from Baseline indicates that FEV1 improved.

Study 009/009e 96-Week Completer Population: participants who completed 48 weeks of treatment with ataluren or placebo in Study 009 and at least 48 weeks of treatment with ataluren in Study 009e. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was validated by using current guidelines of the ATS and ERS. Analyses with the Completer populations were performed to complement the analyses of the Study 009e As-Treated population. Baseline was defined as Week 1 or the most recent value of percent-predicted FEV1 prior to the first dose of open-label treatment in Study 009e.

Study 009/009e 144-Week Completer Population: participants who completed 48 weeks of treatment with ataluren or placebo in Study 009 and at least 96 weeks of treatment with ataluren in Study 009e. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was validated by using current guidelines of the ATS and ERS. The percentage of change in percent-predicted of FEV1 was calculated as follows: ((percent-predicted FEV1-Baseline percent-predicted FEV1)/Baseline percent-predicted FEV1)*100. Analyses with the Completer populations were performed to complement the analyses of the Study 009e As-Treated population. Baseline was defined as Week 1 or the most recent value of percent-predicted FEV1 prior to the first dose of open-label treatment in Study 009e. A positive change from Baseline indicates that FEV1 improved.

Study 009/009e 144-Week Completer Population: participants who completed 48 weeks of treatment with ataluren or placebo in Study 009 and at least 96 weeks of treatment with ataluren in Study 009e. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FVC (the amount of air that can be exhaled after taking a deep breath). Spirometry was validated by using current guidelines of the ATS and ERS. Baseline was defined as Week 1 or the most recent value of percent-predicted FVC prior to the first dose of open-label treatment in Study 009e.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FVC (the amount of air that can be exhaled after taking a deep breath). Spirometry was validated by using current guidelines of the ATS and ERS. The percentage of change in percent-predicted of FVC was calculated as follows: ((percent-predicted FVC-Baseline percent-predicted FVC)/Baseline percent-predicted FVC)*100. Baseline was defined as Week 1 or the most recent value of percent-predicted FVC prior to the first dose of open-label treatment in Study 009e. A positive change from Baseline indicates that FVC improved.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEF25-75 (the rate of air flow during the middle part of an exhalation). Spirometry was validated by using current guidelines of the ATS and ERS. Baseline was defined as Week 1 or the most recent value of percent-predicted FEF25-75 prior to the first dose of open-label treatment in Study 009e.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEF25-75 (the rate of air flow during the middle part of an exhalation). Spirometry was validated by using current guidelines of the ATS and ERS. The percentage of change in percent-predicted of FEF25-75 was calculated as follows: ((percent-predicted FEF25-75-Baseline percent-predicted FEF25-75)/Baseline percent-predicted FEF25-75)*100. Baseline was defined as Week 1 or the most recent value of percent-predicted FEF25-75 prior to the first dose of open-label treatment in Study 009e. A positive change from Baseline indicates that FEF25-75 improved.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

A Respiratory Event Form (REF), which collected data on various signs, symptoms, and effects for each event, was completed by the Investigator when informed by the participant of a respiratory event. Pulmonary exacerbations were assessed by using the modified Fuchs' criteria, which defines an exacerbation as a respiratory event requiring treatment with parenteral antibiotics for any 4 of the following 12 symptoms with or without intravenous (IV) antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; or decrease in pulmonary function by 10% or more from a previously recorded value; or radiographic changes indicative of pulmonary function.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug.

A REF, which collected data on various signs, symptoms, and effects for each event, was completed by the Investigator when informed by the participant of a respiratory event. Pulmonary function was assessed by using the modified Fuchs' criteria, which defines an exacerbation as a respiratory event requiring treatment with parenteral antibiotics for any 4 of the following 12 symptoms with or without treatment with IV antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10% or more from a previously recorded value; or radiographic changes indicative of pulmonary function. The 48-week exacerbation rate was determined by adding the weekly rates for each arm for each 48-week period and dividing the sum by 48.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug.

A REF, which collected data on various signs, symptoms, and effects for each event, was completed by the Investigator when informed by the participant of a respiratory event. Pulmonary function was assessed by using the modified Fuchs' criteria, which defines an exacerbation as a respiratory event requiring treatment with parenteral antibiotics for any 4 of the following 12 symptoms with or without treatment with IV antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10% or more from a previously recorded value; or radiographic changes indicative of pulmonary function. Duration over a 5-week interval is presented. The duration was calculated as follows: estimated date of return to a stable state (as determined by the Investigator) - estimated date of onset of symptoms.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

A REF, which collected data on various signs, symptoms, and effects for each event, was completed by the Investigator when informed by the participant of a respiratory event. Pulmonary function was assessed by using the modified Fuchs' criteria, which defines an exacerbation as a respiratory event requiring treatment with parenteral antibiotics for any 4 of the following 12 symptoms with or without treatment with IV antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10% or more from a previously recorded value; or radiographic changes indicative of pulmonary function. Severity of pulmonary exacerbations over a 5-week interval is presented. The severity of pulmonary exacerbations were graded as mild, moderate, or severe.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

The CFQ-R consists of 44 items, including generic scales of physical functioning, role functioning, vitality, health perceptions, emotional functioning, and social functioning, and CF-specific scales of respiratory and digestive symptoms, body image, eating disturbances, and treatment burden. Questions are scored on a scale from 1 to 4, with higher scores indicating better quality of life (QOL). For some questions, the scale was reversed, so that 1 indicated better QOL. Domain scores were linearly transformed to a 0-100 scale, so that higher scores indicate better QOL. Domain scores were calculated by using the following formula: 100 * (sum of responses - minimum possible sum)/ (maximum possible sum - minimum possible sum). The minimum possible sum = number of questions * 1; the maximum possible = the number of questions * 4. Baseline was Week 1. A negative change from Baseline indicates that health has worsened. Participants may have switched age groups during the study.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for specified categories.

The rate of compliance was defined as the number of actual doses taken divided by the number of planned doses * 100. Participant-reported data were obtained from the participant's compliance log, which was completed by the participant or the caregiver. The participant or caregiver reported how many doses were taken. Compliance by drug accountability was determined by counting used and unused study drug sachets. All calculations were based on the records of the first dose date to the last dose date.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for specified categories.

Blood samples were drawn immediately before administration of the first daily dose (dose taken with breakfast) of ataluren. Whenever possible, the predose sample was to be obtained within 15 minutes of study ataluren administration.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

During treatment, any interventions including hospitalization or use of oral, inhaled, or IV antibiotics was documented if it was due to an exacerbation-like episode. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the Adverse Events module.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug.

During treatment, participants reported when they missed school or work because of pulmonary symptoms. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the Adverse Events module.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug.

During treatment, participants reported when they missed school or work because of pulmonary symptoms. If Event Date was before Day 1 (Baseline) Date, Study Day = Event Date - First Dose Date. If Event Date was on or after Day 1 Date, Study Day = Event Date - First Dose Date + 1. The Duration = Return to Stable Date - Onset Date. Participants with a respiratory event that was ongoing when the participant was discontinued from the study were considered as not evaluable. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the Adverse Events module.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug.

Participants were weighed, and the weight was recorded at Baseline and then every 8 weeks during the treatment period. Baseline was Week 1. A positive change from Baseline indicates that weight increased.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Participants were weighed and measured and the weight and height were recorded at each visit. The BMI was determined by dividing the participant's weight by his or her height. Baseline was Week 1. A positive change from Baseline indicates that BMI increased.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week.

Lungs were imaged by using non-contrast, spiral CT. The administration of CT scans was discontinued for this study via a memorandum sent to all Investigators, based on the results of Study 009, which showed that this exploratory endpoint failed to discriminate active treatment from placebo over the 48-week study period. Therefore, this Outcome Measure was removed from the study as a Secondary Outcome Measure and the CT scans that were administered for this study were not reviewed or analyzed for this Outcome Measure.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number analyzed' signifies the number of participants analyzed for the specified weeks for this Outcome Measure.

TEPD was to be assessed in each nostril by using standardized equipment, techniques, and solutions. Collection of nasal TEPD tracings was discontinued for this study via a memorandum sent to all Investigators, based on the results of Study 009, which showed that this biomarker failed to discriminate active treatment from placebo over the 48-week study period. Therefore, this Outcome Measure was removed from the study as a Secondary Outcome Measure and none of the nasal TEPD tracings were reviewed or analyzed for this Outcome Measure.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number analyzed' signifies the number of participants analyzed for the specified weeks for this Outcome Measure.

Sweat was collected, from each arm, by using pilocarpine iontophoresis. The chloride concentration in the sweat was quantified for each arm by using standard laboratory methods. Tests were considered valid if the sweat collection time was ≤35 minutes; tests with longer collection times were also considered valid if extra time was needed to obtain sufficient volume (≥15uL) for analysis. For analysis purposes, the average of the values from each arm were computed. If the assessment was valid and/or available in only 1 arm, this value was used as if it were the average of both arms. The method used was consistent with the guidelines of the Cystic Fibrosis Foundation Therapeutics - Therapeutic Development Network. Baseline was the most recent value of sweat chloride prior to treatment in Study 009e. A positive change from Baseline indicates that sweat chloride concentration increased.

The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week. One and 4 participants in the ataluren/ataluren and placebo/ataluren groups, respectively, were not evaluable.