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.
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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

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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.

KALYDECO® (ivacaftor) safety profile established in clinical trials

The overall safety profile for KALYDECO is based on Trials 1, 2, and 31

The overall safety profile for KALYDECO is based on pooled data from 3 placebo-controlled clinical trials conducted in 353 patients age 6 years and older with CF who had a G551D mutation in the CFTR gene (Trials 1 and 2) or were homozygous for the F508del mutation (Trial 3)

KALYDECO is not indicated in patients with CF who are homozygous for the F508del mutation

Pooled safety data from Trials 1, 2, and 31,a,b

The proportion of patients who prematurely discontinued study drug due to adverse reactions was 2% for patients treated with KALYDECO and 5% for patients treated with placebo

Serious adverse reactions that occurred more frequently in patients treated with KALYDECO included:

ABDOMINAL PAIN

INCREASED HEPATIC ENZYMES

HYPOGLYCEMIA

The most common adverse reactions in patients treated with KALYDECO in Trials 1, 2, and 3 (N=221) were headache (17%), upper respiratory tract infection (16%), nasal congestion (16%), nausea (10%), rash (10%), rhinitis (6%), dizziness (5%), arthralgia (5%), and bacteria in sputum (5%)

Most common adverse reactions in Trials 1 and 21

MOST COMMON ADVERSE REACTIONS (≥8%) IN PATIENTS WITH A G551D MUTATION TREATED WITH KALYDECO AND GREATER THAN PLACEBO

ADVERSE REACTION
(PREFERRED TERM)

Headache

Oropharyngeal pain

Upper respiratory tract infection

Nasal congestion

Abdominal pain

Nasopharyngitis

Diarrhea

Rash

Nausea

Dizziness

KALYDECO
N=109, n (%)

26 (24)

24 (22)

24 (22)

22 (20)

17 (16)

16 (15)

14 (13)

14 (13)

13 (12)

10 (9)

PLACEBO
N=104, n (%)

17 (16)

19 (18)

14 (14)

16 (15)

13 (13)

12 (12)

10 (10)

7 (7)

11 (11)

1 (1)

The safety profile for the patients with CF enrolled in the other clinical trials (Trials 3-8) was similar to that observed in the 48-week placebo-controlled trials (Trials 1 and 2)c,d,e,f,g


Transaminase elevations in patients age 6 years and older1,a,b

In Trials 1, 2, and 3, the incidence of maximum transaminase (ALT or AST) >8, >5, or >3x ULN was 2%, 2%, and 6% in patients treated with KALYDECO and 2%, 2%, and 8% in patients treated with placebo, respectively

The proportion of patients who permanently discontinued treatment for elevated transaminases, all >8x ULN, was 0.5% for patients treated with KALYDECO and 2% for patients treated with placebo

2 patients treated with KALYDECO were reported to have serious adverse reactions of elevated liver transaminases compared to none on placebo

Transaminase elevations were more common in patients with a history of transaminase elevations


Transaminase elevations in patients age 6 months to less than 6 years1,e,g

In Trial 6, 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

Transaminase elevations were more common in patients who had abnormal transaminases at baseline

KALYDECO was permanently discontinued in 1 patient

In Trial 8, the incidence of patients experiencing transaminase elevations (ALT or AST) >3, >5, and >8x ULN in the cohort of patients age 12 months to less than 24 months was 27.8%, 11.1%, and 11.1%, respectively. In the cohort of patients age 6 months to less than 12 months, 1 patient (9.1%) had elevated ALT of >3 to ≤5x ULN

No patients had elevations in total bilirubin or discontinued treatment due to transaminase elevations

aTrials 1 and 2 were 48-week, Phase 3, randomized, double-blind, placebo-controlled trials in 213 patients with a G551D mutation. Trial 1 patients were 12 years of age and older; Trial 2 patients were 6 to 11 years of age.1

bTrial 3 was a 16-week, randomized, double-blind, placebo-controlled, parallel-group trial in 140 patients with CF age 12 years and older who were homozygous for the F508del mutation. KALYDECO is not effective in patients with CF who are homozygous for the F508del mutation in the CFTR gene.1

cTrial 4 was an 8-week, crossover design trial in 39 patients with a G1244E, G1349D, G178R, G551S, G970R, S1251N, S1255P, S549N, or S549R mutation. Trial 4 patients were between the ages of 6 and 57 years.1

dTrial 5 was a 24-week, placebo-controlled trial in 69 patients with an R117H mutation. Trial 5 patients were between the ages of 6 and 68 years.1

eTrial 6 was a 24-week, open-label trial in 34 patients who could have had a G551D, G1244E, G1349D, G178R, G551S, G970R, S1251N, S1255P, S549N, or S549R mutation. Of the 34 patients enrolled, 32 had the G551D mutation and 2 had the S549N mutation. Trial 6 patients were 2 to less than 6 years of age.1

fTrial 7 was an 8-week crossover design trial involving patients between the ages of 12 and 72 years who were heterozygous for the F508del mutation and a second CFTR mutation predicted to be responsive to ivacaftor. A total of 156 patients were randomized to and received KALYDECO.1

gTrial 8 was a 24-week, open-label trial in a cohort of 19 patients age 12 months to less than 24 months and a cohort of 11 patients age 6 months to less than 12 months, who could have had a G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, or S1255 mutation. Of the 30 patients enrolled, 26 had the G551D mutation (including 1 who had the 2789+5G→A mutation on their second allele), 2 had the S549N mutation, and 1 had the G178R mutation.1-4

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.

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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

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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