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

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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.

12+ YearsTrial 1
12+ YearsTrial 7
TRIAL 1 (STRIVE): Patients with CF age 12 years and older with a G551D mutation
KALYDECO® (ivacaftor) achieved significant improvements across multiple clinical endpoints in patients with a G551D mutation1-3
Mutations eligible for Study1 (mutation in bold was enrolled)

G551D

Trial Design1-3
  • Trial 1 was a 48-week, Phase 3, randomized, double-blind, placebo-controlled trial (N=161) in patients with CF age 12 years and older (mean age: 26 years) and a G551D mutation
  • Patients had to have FEV1 40%-90% predicted at screening [mean FEV1 64% predicted at baseline (range: 32%-98%)]
  • Patients received KALYDECO 150 mg or placebo every 12 hours with fat-containing food, in addition to their prescribed CF therapies. Use of hypertonic saline was not permitted

Primary endpoint1: Improvement in lung function as determined by the mean absolute change from baseline in ppFEV1 through 24 weeks

Other efficacy endpoints1: Absolute change in ppFEV1 through Week 48, improvement from baseline in CFQ-R Respiratory Domain score through Weeks 24 and 48, time to first pulmonary exacerbation through Weeks 24 and 48, absolute change from baseline in body weight at Weeks 24 and 48, and absolute change from baseline in sweat chloride concentration through Weeks 24 and 48

Study Results1,3,4

Significant improvements in lung function were seen through 24 weeks and persisted through 48 weeks1,3,4

Clipboard icon
Treatment difference through:

24 weeks (primary endpoint)a

+10.6 points

(95% CI: 8.6, 12.6; P<.0001)

48 weeks (other endpoint)a

+10.5 points

(95% CI: 8.5, 12.5; P<.0001)

Graph of Absolute Change from Baseline in ppFEV Through Week 48

aTreatment difference = effect of KALYDECO – effect of placebo.1

Primary endpoint was assessed through 24 weeks for Trial 1 and was based on a mixed-effects model for repeated measures (MMRM).1,3


Clinical endpoints studied in Trial 1 for KALYDECO showed significant, sustained improvements vs placebo1

CFQ-R RESPIRATORY DOMAIN SCORE

Treatment difference through:

24 weeksa
+8.1 points
(95% CI: 4.7, 11.4; P<.0001)
48 weeksa
+8.6 points
(95% CI: 5.3, 11.9; P<.0001)

RELATIVE RISK OF PULMONARY EXACERBATION

Treatment difference through:

24 weeksa
60% reduction
(HR, 0.40; P=.0016)
48 weeksa
54% reduction
(HR, 0.46; P=.0012)

  • Pulmonary exacerbation was defined as a change in antibiotic therapy (IV, inhaled, or oral) as a result of 4 or more of 12 prespecified sino-pulmonary signs/symptoms1

BODY WEIGHT

Treatment difference at:

24 weeksa
+2.8 kg
(95% CI: 1.8, 3.7; P<.0001)
48 weeksa
+2.7 kg
(95% CI: 1.3, 4.1; P=.0001)

  • Changes in BMI normalized for age and sex in patients <20 years of age were consistent with absolute change from baseline in weight1

SWEAT CHLORIDE
Treatment difference through:

24 weeksa
-48 mmol/L
(95% CI: -51, -45; P<.0001)
48 weeksa
-48 mmol/L
(95% CI: -51, -45; P<.0001)

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

aTreatment difference = effect of KALYDECO – effect of placebo.1

HR, hazard ratio; IV, intravenous; BMI, body mass index

TRIAL 7: Patients with CF age 12 years and older with specific ivacaftor-responsive mutations
KALYDECO® (ivacaftor) improved lung function vs placebo overall and across all pre-specified subgroups1
Mutations eligible for Study1,2 (mutations in bold were enrolled)

  • Patients were heterozygous for the F508del mutation and a second mutation predicted to be responsive to ivacaftor
  • The 5 splice mutations studied were: 2789+5G→A, 3272-26A→G, 3849+10kbC→T, 711+3A→G, E831X (n=94 for KALYDECO and n=97 for placebo)
  • The 11 missense mutations studied were: A455E, D1152H, D579G, L206W, P67L, R1070W, R117C, R347H, R352Q, S945L, S977F (n=62 for KALYDECO and n=63 for placebo)
  • D110E, D110H, D1270N, E56K, E193K, F1052V, F1074L, K1060T, R74W

Trial Design1,3

CFTR-targeted therapy expanded to patients with responsive splice and missense mutations1

  • Trial 7 was an 8-week, randomized, double-blind, placebo-controlled, 2-period, crossover design trial (N=246) in patients with CF age 12 years and older (mean age: 35 years)a
  • Patients had to have FEV1 40%-90% predicted at screening [mean FEV1 62% predicted at baseline (range: 35%-94%)]
  • Patients received KALYDECO 150 mg or placebo every 12 hours with fat-containing food, in addition to their prescribed CF therapies

Primary endpoint1: Mean absolute change from baseline in ppFEV1 to the average of Weeks 4 and 8

Key secondary endpoint1: Absolute change from baseline in CFQ-R Respiratory Domain score averaged at Weeks 4 and 8


Study Results1,3

Significant improvement in ppFEV1 by Day 15 maintained through 8 weeks1,2


Graph of Absolute Change from Baseline in ppFEV Averaged at Weeks 4 and 8

Adapted with permission from Rowe SM et al. N Engl J Med. 2017;377(21);2024-2035

aThe complete study design and results are not reported here; only the KALYDECO and placebo groups are shown

Improvements in ppFEV1 were observed with KALYDECO® across all pre-specified subgroups vs placebo3,4,b,c

Chart depicting Changes in ppFEV, by Subgroup

bAdditional subgroups analyzed included: region; sex; colonization of Pseudomonas aeruginosa; use of azithromycin; and use of inhaled antibiotics, bronchodilators, hypertonic saline, and corticosteroids.

cTreatment difference = effect of KALYDECO – effect of placebo.1

  • For individual mutations, changes in ppFEV1 varied by genotype and ranged from +2.4% points to +13.3% points; see the full Prescribing Information for results by mutation. These were ad hoc analyses1

Significant improvement in CFQ-R Respiratory Domain score from baseline for KALYDECO vs placebo overall1,2

  • The overall treatment difference was 9.7 points (95% CI: 7.2, 12.2; P<.0001)
  • For individual mutations, results varied by genotype and ranged from -8.3 to 44.4; see full Prescribing Information for results by mutation. These were ad hoc analyses
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

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.

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

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.

minus icon
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

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.

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

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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