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

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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 Years to 11 YearsTrial 2
6+ YearsTrial 4
6+ YearsTrial 5
TRIAL 2 (ENVISION): Patients with CF age 6 to 11 with G551D mutation
KALYDECO® (ivacaftor): Efficacy results include significant improvement in lung function1,2
Mutations Eligible for Study1 (mutation in bold was enrolled)

G551D

Trial Design1
  • Trial 2 was a 48-week, Phase 3, randomized, double-blind, placebo-controlled trial (N=52) in patients with CF age 6 to 11 years (mean age: 9 years) and a G551D mutation
  • Patients had to have FEV1 40%-105% predicted at screening [mean FEV1 84% predicted at baseline (range: 44%-134%)]
  • 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 Week 24

Other efficacy endpoints1: Absolute change in ppFEV1 through Week 48, improvement from baseline in CFQ-R Respiratory Domain score 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 Results1,2

Improvements in ppFEV1 vs placebo were seen at the first post-baseline visit and persisted through 48 weeks1,2

Treatment difference through:

24 weeks (primary endpoint)1,2,a

+12.5 points

(95% CI: 6.6, 18.3; P <.0001)

48 weeks (secondary endpoint)2,a

+10.0 points

(95% CI: 4.5, 15.5; P <.001)

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 2 and was based on a mixed-effects model for repeated measures (MMRM).1,2

CFQ-R, Cystic Fibrosis Questionnaire-Revised; SEM, standard error of the mean.

Other efficacy endpoints1

CFQ-R RESPIRATORY DOMAIN SCORE

Treatment difference through:

24 weeksa
+6.1 points
(95% CI: -1.4, 13.5; not statistically significant)
48 weeksa
+5.1 points
(95% CI: -1.6, 11.8; not statistically significant)

  • CFQ-R Respiratory Domain score evaluated relevant CF respiratory symptoms, including cough, sputum production, and difficulty breathing1

BODY WEIGHT

Treatment difference at:

24 weeksa
+1.9 kg
(95% CI: 0.9, 2.9; P=.0004)
48 weeksa
+2.8 kg
(95% CI: 1.3, 4.2; P=.0002)

SWEAT CHLORIDE (pharmacodynamic measure)

Treatment difference through:

24 weeksa
-54 mmol/L
(95% CI: -62, -47; P <.0001)
48 weeksa
-53 mmol/L
(95% CI: -61, -46; 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

The risk of pulmonary exacerbations was not analyzed in Trial 2 due to low incidence of events.1

TRIAL 4 (KONNECTION): Patients with CF age 6 years and older
KALYDECO® (ivacaftor): Significant improvements demonstrated for the overall population with eligible mutations1,2
Mutations Eligible for Study1 (mutations in bold were enrolled)

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

*Based on the clinical and pharmacodynamic (sweat chloride) responses to ivacaftor, efficacy in patients with the G970R mutation could not be established.

Trial Design1
  • Trial 4 was a Phase 3, two-part, randomized, double-blind, placebo-controlled, crossover design trial (two 8-week treatment periods separated by a 4- to 8-week washout period; N=39) in patients with CF age 6 years and older (mean age: 23 years)
  • Patients had either a G1244E, G1349D, G178R, G551S, G970R, S1251N, S1255P, S549N, or S549R mutation
  • KALYDECO is not indicated for use in patients with a G970R mutation
  • Patients had to have FEV1 ≥40% predicted at screening [mean FEV1 78% predicted at baseline (range: 43%-119%)]
  • Patients received KALYDECO 150 mg or placebo every 12 hours with fat-containing food, in addition to their prescribed CF therapies

Primary endpoint1: Improvement in lung function as determined by the mean absolute change from baseline in ppFEV1 through 8 weeks

Other endpoints1: Absolute change from baseline in: body mass index (BMI) at 8 weeks, improvement in CFQ-R Respiratory Domain score through 8 weeks, and sweat chloride concentration through 8 weeks

Study Results2
There was a high degree of variability of efficacy responses among the 9 mutations studied1

For more information, please see the full Prescribing Information.

Significant improvements in ppFEV1 were maintained through 8 weeks of treatment2

Graph of Absolute Change from Baseline in ppFEV
  • For individual mutations, mean increases in ppFEV1 ranged from +3% points to +20% points at Week 8
  • Based on the clinical and pharmacodynamic (sweat chloride) responses to ivacaftor, efficacy in patients with the G970R mutation could not be established
There was a high degree of variability of responses among the 9 mutations studied1,2
BMI
Treatment difference at 8 weeksa
+0.66 kg/m2
(95% CI: 0.34, 0.99; P<.0001)
  • Mean increases ranged from +0.16 kg/m2 to +1.62 kg/m2* at Week 8
CFQ-R RESPIRATORY DOMAIN SCORE
Treatment difference through 8 weeksa
+9.6 points
(95% CI: 4.5, 14.7; P=.0004)
  • CFQ-R Respiratory Domain score evaluated relevant CF respiratory symptoms, including cough, sputum production, and difficulty breathing
  • Mean increases ranged from +1.4 points to +23.3 points at Week 8
SWEAT CHLORIDE1,2
Treatment difference through 8 weeksa
-49 mmol/L
(95% CI: -57, -41; P<.0001)
  • There was no direct correlation between decrease in sweat chloride levels and improvement in lung function (FEV1)
  • Reductions ranged from –80 mmol/L to -6 mmol/L at Week 8

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

*Reflects results from the 1 patient with the G551S mutation with data at the 8-week time point.

See the full Prescribing Information for complete results by mutation.

TRIAL 5 (KONDUCT): Patients with CF age 6 years and older
KALYDECO® (ivacaftor) in patients with the R117H mutation1,2
Mutations Eligible for Study1 (mutation in bold was enrolled)

R117H

Trial Design1
  • Trial 5 was a 24-week, Phase 3, randomized, double-blind, placebo-controlled, parallel-group trial in patients with CF age 6 years and older (mean age: 31 years) who had an R117H mutation
  • Patients age 12 years and older had to have FEV1 40%-90% predicted at screening; patients age 6 to 11 years had to have FEV1 40%-105% predicted at screening; mean FEV1 73% predicted at baseline (range: 33%-106%)
  • Patients received KALYDECO 150 mg or placebo every 12 hours with fat-containing food, in addition to their prescribed CF therapies
  • Subgroups analyzed were based on age, lung function, and poly-T status

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 body mass index (BMI) at Week 24, CFQ-R Respiratory Domain score through Week 24, time to first pulmonary exacerbation, and absolute change in sweat chloride from baseline through Week 24


Study Results1

Overall change in ppFEV1 through Week 24, 2.1 percentage points (N=69), was not statistically significant1


Chart depicting Absolute Change in ppFEV, Through Week 24

aMMRM analysis with fixed effects for treatment, age, week, baseline value, treatment by week, and subject as a random effect.
bPoly-T status confirmed by genotyping (n=54).

Results for other efficacy endpoints studied in Trial 51,2
BODY WEIGHT
Treatment difference at 24 weeksa
+0.3 kg/m2
(95% CI: -1.57, 2.10; not statistically significant)
CFQ-R RESPIRATORY DOMAIN SCORE
Treatment difference through 24 weeksa
+8.4 points
(95% CI: 2.2, 14.6; P=.009*)
*p-values are nominal
  • CFQ-R Respiratory Domain score evaluated relevant CF respiratory symptoms, including cough, sputum production, and difficulty breathing
  • Results ranged from –6.1 to +15.3 points
RELATIVE RISK OF PULMONARY EXACERBATION
Treatment difference through 24 weeksa
7% reduction
(HR, 0.93; not statistically significant)
SWEAT CHLORIDE
(pharmacodynamic measure)1,2
Treatment difference through 24 weeksa
-24 mmol/L
(95% CI: -28.0, -19.9; P<.0001*)
*p-values are nominal
  • There was no direct correlation between decrease in sweat chloride levels and improvement in lung function (FEV1)
  • Reductions in sweat chloride were seen in all subgroups, and ranged from –27.6 mmol/L to –20.0 mmol/L

HR, hazard ratio; BMI, body mass index

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

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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