KALYDECO® (ivacaftor) is now indicated to treat people age 6 months and older with one of 38 CFTR mutations1
KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 6 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
Patient eligibility mutation chart
for KALYDECO

About KALYDECO® (ivacaftor)

Overview of Studies and Data

Age 6 Months to Less Than 6 Years

Age 6 Years and Older

Age 12 Years and Older

In Vitro Results

Safety Profile

Dosing

Administration

Drug Interactions

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Indications and Usage

KALYDECO® (ivacaftor) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 6 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.

6 Months to <2 YearsTrial 8
2 Years to <6 YearsTrial 6
TRIAL 8 (ARRIVAL): Patients with CF age 6 months to less than 2 years
KALYDECO® (ivacaftor) in patients as young as 6 months of age1,2

Not all indicated genotypes were evaluated in each study; additionally, the below is not representative of all endpoints evaluated in each study.

Trial Design1-4
  • Trial 8 was a Phase 3, 24-week, open-label, 2-part study of KALYDECO that included the following cohorts of patients with CF: Patients age 6 to <12 months (n=11) and patients age 12 to <24 months (n=19)1,3
  • Patients were eligible if they had a G551D, G1244E, G1349D, G178R, G551S, R117H*, S1251N, S1255P, S549N, or S549R mutation1,2-4 (mutations in bold were enrolled)
  • 6 to <12 months cohort: Enrolled patients had either a G551D mutation (n=10) or a G178R (n=1) mutation2
  • 12 to <24 months cohort: Enrolled patients had the G551D mutation (n=16), S549N mutation (n=2), or G178R mutation (n=1)3
  • Patients received KALYDECO every 12 hours with fat-containing food, in addition to their prescribed CF therapies, based on weight: 5 kg to <7 kg: One 25-mg packet of oral granules; 7 kg to <14 kg: One 50-mg packet of oral granules; ≥14 to <25 kg: One 75-mg packet of oral granules2,3

Primary endpoint2,3: Safety, assessed by adverse events and clinical laboratory assessments

Secondary endpoint2,3: Absolute change from baseline in sweat chloride concentration at 24 weeks

*Patients with the R117H mutation were only eligible to enroll in this study in the US.

Trial 8 limitations2,3
  • The study was open label and not placebo controlled; therefore, causality cannot be attributed
  • All patients in the study knew they were on active drug, which may have introduced a treatment bias
Discontinuations and serious adverse events2
  • 6 to <12 month cohort: There was 1 treatment interruption and no permanent discontinuations due to adverse events. Serious adverse events included viral respiratory tract infection (1), viral rash (1), and cough (1)2
  • 12 to <24 month cohort: 1 patient discontinued due to withdrawal of consent. 4 serious adverse events occurred in 2 patients3
  • – 1 patient had constipation, distal intestinal obstruction syndrome (DIOS), and eczema herpeticum
  • – 1 patient had persistent cough

The serious adverse event of constipation was considered possibly related to ivacaftor by the investigator. All other serious adverse events were considered unrelated or unlikely to be related to ivacaftor by the investigator.

The safety profile of patients in this trial is similar to that observed in patients age 2 years and older.1
Transaminase elevations1,3,5
incidence of transaminase elevations

ELEVATED ALT OR AST

>3x ULN

>5x ULN

>8x ULN

6 to <12 MONTH COHORT
n/N (%)

1/11 (9.1)

0

0

12 to <24 MONTH COHORT
n/N (%)

5/18 (27.8)

2/18 (11.1)

2/18 (11.1)

ALT, alanine aminotransferase. AST, aspartate aminotransferase. ULN, upper limit of normal.

Please see Important Safety Information for additional information on liver-related serious adverse events.

  • No patients in either cohort had elevations in total bilirubin, or discontinued ivacaftor treatment due to transaminase elevations3
  • 6 to <12 month cohort: During the course of this study, no patient interrupted treatment because of adverse events of elevated liver function tests2
  • – On the last day of the study, 1 patient had an LFT elevation that resulted in subsequent treatment interruption
  • 12 to <24 month cohort: 2 patients had study drug interrupted because of adverse events of elevated liver function tests3
  • 1 patient had adverse events of ALT increased and AST increased that led to study drug interruption5
  • 1 patient had adverse events of ALT increased, AST increased, and GGT increased that led to study drug interruption5
  • Both resumed treatment following a period of study drug interruption with no further elevations3

Please see Important Safety Information for more information about transaminase elevations reported in patients with CF receiving KALYDECO.

Study Results1,3

Pharmacodynamic results1,2
Reduction in sweat chloride from baseline with KALYDECO: Patients age 6 to less than 12 months

bMean (SD) concentrations by visit calculated from the number of children contributing data at each time point.

cCalculated from children with data available at both baseline and Week 24.

  • In the 6 to <12 month cohort (n=6), the mean absolute change from baseline in sweat chloride was –58.6 mmol/L (95% CI: –75.9, –41.3) at Week 24 (secondary endpoint)1,2

  • In clinical studies with KALYDECO, there was no direct correlation between decrease in sweat chloride levels and improvement in lung function (FEV1)1

The safety and efficacy of KALYDECO in patients with CF younger than 6 months of age have not been established. The use of KALYDECO in children under the age of 6 months is not recommended.1
Reduction in sweat chloride from baseline with KALYDECO: Patients age 12 months to less than 24 months
Graph of Mean Sweat Chloride Concentrations By Visit

dMean (SD) concentrations by visit calculated from the number of children contributing data at each time point.

eCalculated from children with data available at both baseline and Week 24.

  • In the 12 month to <24 month cohort (n=10), the mean absolute change from baseline in sweat chloride for patient was −73.5 mmol/L (95% CI: −86.0, −61.0) at Week 24 (secondary endpoint)1,3

  • In clinical studies with KALYDECO, there was no direct correlation between decrease in sweat chloride levels and improvement in lung function (FEV1)1

CI, confidence interval; SD, standard deviation.

TRIAL 6 (KIWI): Patients with CF age 2 to less than 6 years
KALYDECO® (ivacaftor): Safety consistent with patients age 6 years and older1,2
Mutations Eligible for Study2 (mutations in bold were enrolled)

G551D, G1244E, G1349D, G178R, G551S, G970R*, S1251N, S1255P, S549N, or S549R

*KALYDECO is not indicated in use for patients with a G970R mutation1

Trial Design1,2

• Trial 6 was a 24-week, open-label trial (N=34) evaluating safety, pharmacokinetics, and pharmacodynamics in patients with CF age 2 to less than 6 years (mean age: 3 years)1

  • Patients were eligible if they had either a G551D, G1244E, G1349D, G178R, G551S, G970R*, S1251N, S1255P, S549N, or S549R mutation2
  • Of the 34 patients enrolled, 32 had the G551D mutation and 2 had the S549N mutation2

  • Patients received KALYDECO every 12 hours with fat-containing food, in addition to their prescribed CF therapies, based on weight; 7 kg to < 14 kg: One 50-mg packet of oral granules; ≥14 kg to < 25 kg: One 75-mg packet of oral granules2

Primary outcome2: Safety, assessed by adverse events and clinical laboratory assessments

Select secondary outcome measure2: Absolute change from baseline in sweat chloride concentration at 24 weeks

Trial 6 limitations2
  • The study was open label and not placebo controlled; therefore, causality cannot be attributed
  • All patients in the study knew they were on active drug, which may have introduced a treatment bias
Study Results1

Safety results and pharmacokinetics were similar to older patients1

  • The safety profile for patients in Trial 6, including type and frequency of adverse reactions, was similar to that observed in Trials 1 and 2
  • Transaminase elevations were more common in patients who had abnormal transaminases at baseline
  • The incidence of patients experiencing transaminase elevations (ALT or AST) >3x ULN was 14.7% (5/34). All 5 patients had maximum ALT or AST levels >8x ULN, which returned to baseline levels following interruption of KALYDECO dosing. KALYDECO was permanently discontinued in 1 patient
  • Ivacaftor exposure in patients age 2 to less than 6 years administered either 50 mg or 75 mg of KALYDECO oral granules every 12 hours was similar to that observed in older patients administered KALYDECO tablets
  • KALYDECO oral granules (2 x 75 mg) had similar bio-availability as the 150-mg tablet when given with fat-containing food
The efficacy of KALYDECO in patients age 2 to less than 6 years was extrapolated from patients age 6 years and older with support from population pharmacokinetic analyses showing similar drug exposure levels in adults and children age 2 to less than 6 years.1
Reduction in sweat chloride from baseline (pharmacodynamic measure) with KALYDECO (ivacaftor)1,*
Graph of Mean Sweat Chloride Concentrations By Visit

*SD 24.3, P=.002 for patients who received 50 mg; SD 27.6, P<.0001 for patients who received 75 mg.2

  • The mean absolute change from baseline in sweat chloride for patients age 2 to less than 6 years (n=34) was –45 mmol/L (95% CI: –53, –38) through Week 24 (secondary endpoint)1,3

  • There was no direct correlation between decrease in sweat chloride levels and improvement in lung function (FEV1)1

CI, confidence interval; SD, standard deviation.

Important Safety Information
Transaminase (ALT or AST) Elevations

Elevated transaminases have been reported in patients with CF receiving KALYDECO. Transaminase elevations were more common in patients with a history of transaminase elevations or in patients who had abnormal transaminases at baseline. It is recommended that ALT and AST be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. For patients with a history of transaminase elevations, more frequent monitoring of liver function tests should be considered

Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN). Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO dosing

Indications and Usage

KALYDECO® (ivacaftor) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 6 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.

If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

Concomitant Use With CYP3A Inducers

Use of KALYDECO with strong CYP3A inducers, such as rifampin, substantially decreases the exposure of ivacaftor, which may reduce the therapeutic effectiveness of KALYDECO. Co-administration of KALYDECO with strong CYP3A inducers, such as rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, and St. John’s wort is not recommended

Cataracts

Cases of non-congenital lens opacities/cataracts have been reported in pediatric patients treated with KALYDECO. Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating KALYDECO treatment

Pediatric Use

The safety and efficacy of KALYDECO in patients with CF younger than 6 months of age have not been studied. The use of KALYDECO in children under the age of 6 months is not recommended

Serious Adverse Reactions

Serious adverse reactions, whether considered drug-related or not by the investigators, which occurred more frequently in patients treated with KALYDECO included abdominal pain, increased hepatic enzymes, and hypoglycemia

Most Common Adverse Reactions

The most common adverse reactions in patients with a G551D mutation in the CFTR gene (Trials 1 and 2) with an incidence of ≥8% and at a higher incidence for patients treated with KALYDECO (N=109) than for placebo (N=104) were headache, oropharyngeal pain, upper respiratory tract infection, nasal congestion, abdominal pain, nasopharyngitis, diarrhea, rash, nausea, and dizziness

The safety profiles for patients with additional approved mutations enrolled in Trials 4, 5, and 7; and for patients ages 2 to less than 6 years enrolled in Trial 6; and for patients aged 6 months to less than 24 months enrolled in Trial 8; were similar to that observed in Trials 1 and 2

Click here to access full Prescribing Information for KALYDECO (ivacaftor).

Reference: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. The Clinical and Functional Translation of CFTR (CFTR2); available at http://cftr2.org. Accessed April 8, 2019. 3. Cystic Fibrosis Genetic Analysis Consortium, The Hospital for Sick Children. Cystic Fibrosis Mutation Database (CFTR1). http://www. genet.sickkids.on.ca/app. Accessed April 8, 2019. 4. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. REF-2303; 2019.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Davies JC, Wang LT, Campbell D, et al. Ivacaftor treatment in patients 6 to <12 months old with a CFTR gating mutation: results of a Phase 3, two-part, single-arm study. Poster and abstract presented at: North American Cystic Fibrosis Conference; October 2018; Denver, CO. 3. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018; 6(7):545-553. 4. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018; 6(7)(suppl):545-553. 5. Davies JC, Cunningham S, Harris WT, et al. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016;4(2):107-115. 6. Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with G551D mutation. Am J Respir Crit Care Med. 2013;187(11):1219–1225. 7. De Boeck K, Munck A, Walker S, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014;13(6):674-680. 8. Moss RB, Flume PA, Elborn JS, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomized controlled trial. Lancet Respir Med. 2015;3(7):524-533. 9. US National Library of Medicine. ClinicalTrials.gov. Available at https://clinicaltrials.gov. Accessed April 8, 2019. 10. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1691. 11. Rowe SM, Daines C, Ringshausen FC, et al. Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med. 2017;377(21)(suppl):2024-2035. 12. Van Goor F, Yu H, Burton B, Hoffman BJ. Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros. 2014;13(1):29-36.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Davies JC, Wang LT, Campbell D, et al. Ivacaftor treatment in patients 6 to <12 months old with a CFTR gating mutation: results of a Phase 3, two-part, single-arm study. Poster and abstract presented at: North American Cystic Fibrosis Conference; October 2018; Denver, CO. 3. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018;6(7):545-553. 4. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018; 6(7)(suppl):553. 5. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00198; 2018.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Davies JC, Cunningham S, Harris WT, et al. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016;4(2):107-115. 3. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00239; 2018.

References: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with G551D mutation. Am J Respir Crit Care Med. 2013;187(11):1219–1225.

References: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. De Boeck K, Munck A, Walker S, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014;(6)13:674-680.

References: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Moss RB, Flume PA, Elborn JS, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomized controlled trial. Lancet Respir Med. 2015;3(7):524-533.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. US National Library of Medicine. ClinicalTrials.gov. Available at https://clinicaltrials.gov. Accessed April 8, 2019. 3. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672. 4. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18)(suppl):1663-1672.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Rowe SM, Daines C, Ringshausen FC, et al. Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med. 2017;377(21)(suppl):2024-2035. 3. Rowe SM, Daines, C Ringshausen FC, et al. Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med. 2017;377(21);2024-2035. 4. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00197; 2018.

References: 1. Van Goor F, Yu H, Burton B, Hoffman BJ. Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros. 2014;13(1):29-36. 2. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 3. Sosnay P, et al. Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene. Nat Genet. 2013;45(10):1160-1167. 4. FDA approves ivacaftor for 23 additional CFTR mutations [press release]. Cystic Fibrosis Foundation; May 17, 2017.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018;6(7):545-553. 3. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018;6(7)(suppl):545-553. 4. Davies JC, Wang LT, Campbell D, et al. Ivacaftor treatment in patients 6 to <12 months old with a CFTR gating mutation: results of a Phase 3, two-part, single-arm study. Poster and abstract presented at: North American Cystic Fibrosis Conference; October 2018; Denver, CO.

Reference: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019.

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Important Safety Information
Transaminase (ALT or AST) Elevations

Elevated transaminases have been reported in patients with CF receiving KALYDECO. Transaminase elevations were more common in patients with a history of transaminase elevations or in patients who had abnormal transaminases at baseline. It is recommended that ALT and AST be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. For patients with a history of transaminase elevations, more frequent monitoring of liver function tests should be considered

Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN). Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO dosing

Indications and Usage

KALYDECO® (ivacaftor) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 6 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.

If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

Concomitant Use With CYP3A Inducers

Use of KALYDECO with strong CYP3A inducers, such as rifampin, substantially decreases the exposure of ivacaftor, which may reduce the therapeutic effectiveness of KALYDECO. Co-administration of KALYDECO with strong CYP3A inducers, such as rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, and St. John’s wort is not recommended

Cataracts

Cases of non-congenital lens opacities/cataracts have been reported in pediatric patients treated with KALYDECO. Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating KALYDECO treatment

Pediatric Use

The safety and efficacy of KALYDECO in patients with CF younger than 6 months of age have not been studied. The use of KALYDECO in children under the age of 6 months is not recommended

Serious Adverse Reactions

Serious adverse reactions, whether considered drug-related or not by the investigators, which occurred more frequently in patients treated with KALYDECO included abdominal pain, increased hepatic enzymes, and hypoglycemia

Most Common Adverse Reactions

The most common adverse reactions in patients with a G551D mutation in the CFTR gene (Trials 1 and 2) with an incidence of ≥8% and at a higher incidence for patients treated with KALYDECO (N=109) than for placebo (N=104) were headache, oropharyngeal pain, upper respiratory tract infection, nasal congestion, abdominal pain, nasopharyngitis, diarrhea, rash, nausea, and dizziness

The safety profiles for patients with additional approved mutations enrolled in Trials 4, 5, and 7; and for patients ages 2 to less than 6 years enrolled in Trial 6; and for patients aged 6 months to less than 24 months enrolled in Trial 8; were similar to that observed in Trials 1 and 2

Click here to access full Prescribing Information for KALYDECO (ivacaftor).

Reference: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. The Clinical and Functional Translation of CFTR (CFTR2); available at http://cftr2.org. Accessed April 8, 2019. 3. Cystic Fibrosis Genetic Analysis Consortium, The Hospital for Sick Children. Cystic Fibrosis Mutation Database (CFTR1). http://www. genet.sickkids.on.ca/app. Accessed April 8, 2019. 4. Cystic Fibrosis Foundation Patient Registry. 2017 Annual Data Report. Bethesda, Maryland. ©2018 Cystic Fibrosis Foundation.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Davies JC, Wang LT, Campbell D, et al. Ivacaftor treatment in patients 6 to <12 months old with a CFTR gating mutation: results of a Phase 3, two-part, single-arm study. Poster and abstract presented at: North American Cystic Fibrosis Conference; October 2018; Denver, CO. 3. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018; 6(7):545-553. 4. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018; 6(7)(suppl):545-553. 5. Davies JC, Cunningham S, Harris WT, et al. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016;4(2):107-115. 6. Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with G551D mutation. Am J Respir Crit Care Med. 2013;187(11):1219–1225. 7. De Boeck K, Munck A, Walker S, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014;13(6):674-680. 8. Moss RB, Flume PA, Elborn JS, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomized controlled trial. Lancet Respir Med. 2015;3(7):524-533. 9. US National Library of Medicine. ClinicalTrials.gov. Available at https://clinicaltrials.gov. Accessed April 8, 2019. 10. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1691. 11. Rowe SM, Daines C, Ringshausen FC, et al. Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med. 2017;377(21)(suppl):2024-2035. 12. Van Goor F, Yu H, Burton B, Hoffman BJ. Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros. 2014;13(1):29-36.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Davies JC, Wang LT, Campbell D, et al. Ivacaftor treatment in patients 6 to <12 months old with a CFTR gating mutation: results of a Phase 3, two-part, single-arm study. Poster and abstract presented at: North American Cystic Fibrosis Conference; October 2018; Denver, CO. 3. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018;6(7):545-553. 4. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018; 6(7)(suppl):553. 5. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00198; 2018.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Davies JC, Cunningham S, Harris WT, et al. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016;4(2):107-115. 3. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00239; 2018.

References: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with G551D mutation. Am J Respir Crit Care Med. 2013;187(11):1219–1225.

References: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. De Boeck K, Munck A, Walker S, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014;(6)13:674-680.

References: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Moss RB, Flume PA, Elborn JS, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomized controlled trial. Lancet Respir Med. 2015;3(7):524-533.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. US National Library of Medicine. ClinicalTrials.gov. Available at https://clinicaltrials.gov. Accessed April 8, 2019. 3. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672. 4. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18)(suppl):1663-1672.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Rowe SM, Daines C, Ringshausen FC, et al. Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med. 2017;377(21)(suppl):2024-2035. 3. Rowe SM, Daines, C Ringshausen FC, et al. Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med. 2017;377(21);2024-2035. 4. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00197; 2018.

References: 1. Van Goor F, Yu H, Burton B, Hoffman BJ. Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros. 2014;13(1):29-36. 2. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 3. Sosnay P, et al. Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene. Nat Genet. 2013;45(10):1160-1167. 4. FDA approves ivacaftor for 23 additional CFTR mutations [press release]. Cystic Fibrosis Foundation; May 17, 2017.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019. 2. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018;6(7):545-553. 3. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018;6(7)(suppl):545-553. 4. Davies JC, Wang LT, Campbell D, et al. Ivacaftor treatment in patients 6 to <12 months old with a CFTR gating mutation: results of a Phase 3, two-part, single-arm study. Poster and abstract presented at: North American Cystic Fibrosis Conference; October 2018; Denver, CO.

Reference: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019.

References: 1. KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; April 2019.

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