Ivacaftor

Ivacaftor is a potentiator of the CFTR protein. The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs.

• KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . 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.

• None.contraindicated

Age Weight Dosage Administration 1 month to less than 2 months 3 kg or greater One 5.8 mg packet every 12 hours Mixed with one teaspoon (5 mL) of soft food or liquid and administered orally with fat-containing food 2 months to less than 4 months 3 kg or greater One 13.4 mg packet every 12 hours 4 months to less than 6 months 5 kg or greater One 25 mg packet every 12 hours 6 months to less than 6 years 5 kg to less than 7 kg One 25 mg packet every 12 hours 7 kg to less than 14 kg One 50 mg packet every 12 hours 14 kg or greater One 75 mg packet every 12 hours 6 years and older - One 150 mg tablet every 12 hours Taken orally with fat-containing food See full prescribing information for the recommended dosage in patients aged 6 months and older with moderate or severe hepatic impairment. ( 2.3 , 8.6 ) See full prescribing information for dosage modifications due to drug interactions with KALYDECO. ( 2.4 , 7.1 ) Not recommended in pediatric patients less than 1 month of age. ( 2.2 , 8.4 ) Not recommended in patients 1 month to less than 6 months of age with any level of hepatic impairment and/or taking concomitant moderate or strong CYP3A inhibitors. ( 2.3 , 2.4 , 8.6 ) 2.1 Recommended Dosage in Adults and Pediatric Patients Aged 6 Years and Older The recommended dosage of KALYDECO for adults and pediatric patients aged 6 years and older is 150 mg orally every 12 hours (300 mg total daily dose) with fat-containing food [ see Dosage and Administration (2.5) ].

Elevated transaminases (ALT or AST): Transaminases (ALT and AST) should be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. In patients with a history of transaminase elevations, more frequent monitoring of liver function tests should be considered. Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Interrupt dosing 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. ( 5.1 , 6 ) Hypersensitivity reactions: Anaphylaxis has been reported with KALYDECO in the postmarketing setting. Initiate appropriate therapy in the event of a hypersensitivity reaction. ( 5.2 ) Intracranial hypertension : Intracranial hypertension (IH) has been reported in the postmarketing setting with use of drugs containing the same or similar active ingredients as KALYDECO. If an unusual headache or visual disturbances occur during treatment, and IH is suspected, interrupt KALYDECO and refer for prompt medical evaluation. ( 5.3 ) Neuropsychiatric events, including suicidal thoughts and behaviors : Serious neuropsychiatric events, including symptoms of anxiety, depression, suicidal ideation and behavior, and sleep disturbances, have been reported in the postmarketing setting for KALYDECO or drugs containing the same or similar active ingredient. Monitor patients closely for new or worsening symptoms.

The following adverse reactions are discussed in greater detail in other sections of the labeling: Transaminase Elevations [ see Warnings and Precautions (5.1) ] Hypersensitivity Reactions, Including Anaphylaxis [see Warnings and Precautions (5.2) ] Intracranial Hypertension [see Warnings and Precautions (5.3) ] Neuropsychiatric Events, Including Suicidal Thoughts and Behaviors [see Warnings and Precautions (5.4) ] Cataracts [see Warnings and Precautions (5.6) ] The most common adverse drug reactions to KALYDECO (≥8% of patients with CF who have a G551D mutation in the CFTR gene) were headache, oropharyngeal pain, upper respiratory tract infection, nasal congestion, abdominal pain, nasopharyngitis, diarrhea, rash, nausea, and dizziness. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Vertex Pharmaceuticals Incorporated at 1-877-634-8789 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice. The overall safety profile of KALYDECO is based on pooled data from three placebo-controlled clinical trials conducted in 353 patients 6 years of age 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).

8.1 Pregnancy Risk Summary There are limited and incomplete human data from clinical trials and postmarketing reports on use of KALYDECO in pregnant women. In animal reproduction studies, oral administration of ivacaftor to pregnant rats and rabbits during organogenesis demonstrated no teratogenicity or adverse effects on fetal development at doses that produced maternal exposures up to approximately 5 (rats) and 11 (rabbits) times the exposure at the maximum recommended human dose (MRHD). No adverse developmental effects were observed after oral administration of ivacaftor to pregnant rats from organogenesis through lactation at doses that produced maternal exposures approximately 3 times the exposures at the MRHD, respectively ( see Data ). The background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects is 2% to 4% and miscarriage is 15% to 20% in clinically recognized pregnancies. Data Animal Data In an embryo-fetal development study, pregnant rats were administered ivacaftor at oral doses of 50, 100, or 200 mg/kg/day during the period of organogenesis from gestation days 7-17. Ivacaftor did not affect fetal survival at exposures up to 5 times the MRHD (based on summed AUCs for ivacaftor and its metabolites at maternal oral doses up to 200 mg/kg/day).

Metabolism

The pharmacokinetics of ivacaftor is similar between healthy adult volunteers and patients with CF. After oral administration of a single 150 mg dose to healthy volunteers in a fed state, peak plasma concentrations (T max ) occurred at approximately 4 hours, and the mean (±SD) for AUC and C max were 10600 (5260) ng*hr/mL and 768 (233) ng/mL, respectively.

There have been no reports of overdose with KALYDECO. No specific antidote is available for overdose with KALYDECO. Treatment of overdose with KALYDECO consists of general supportive measures including monitoring of vital signs and observation of the clinical status of the patient.

• Jan 31, 2012NDANDA203188Vertex Pharms
• Mar 17, 2015NDANDA207925Vertex Pharms Inc
• Jul 2, 2015NDANDA206038Vertex Pharms Inc
• Feb 12, 2018NDANDA210491Vertex Pharms Inc
• Aug 7, 2018NDANDA211358Vertex Pharms Inc
• Oct 21, 2019NDANDA212273Vertex Pharms Inc
• Apr 26, 2023NDANDA217660Vertex Pharms Inc

1. 1Infective Pulmonary Exacerbation Of Cystic Fibrosis4,02712%
2. 2Hospitalisation3,0899.2%
3. 3Cough1,3764.1%
4. 4Headache1,3113.9%
5. 5Infection1,3063.9%
6. 6Cystic Fibrosis1,2953.9%
7. 7Pneumonia1,1903.5%
8. 8Dyspnoea1,1283.4%
9. 9Fatigue9162.7%
10. 10Pulmonary Function Test Decreased8812.6%
11. 11Anxiety8062.4%
12. 12Abdominal Pain Upper7962.4%
13. 13Malaise7552.2%
14. 14Rash7512.2%
15. 15Nausea7362.2%

The 10 most recently updated of 199 ClinicalTrials.gov registrations naming Ivacaftor as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Pharmacokinetics of Antibiotics in Patients With Cystic Fibrosis Trated With Elexacaftor/Tezacaftor/Ivacaftor (ETI)
  Recruiting · Observational · 30 enrolled · Fondation Ildys
  NCT07629986updated 2026-06-05
• Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older
  Active not recruiting · Phase 3 · Interventional · 50 enrolled · Vertex Pharmaceuticals Incorporated
  NCT06460506updated 2026-06-05
• Ensuring Access to Optimal Therapy in CF: The ENACT Study
  Recruiting · Phase 4 · Interventional · 100 enrolled · Arkansas Children's Hospital Research Institute
  NCT07148739updated 2026-06-02
• Glucose Metabolism in Cystic Fibrosis Related Diabetes (CFRD)
  Recruiting · Observational · 30 enrolled · University of Alabama at Birmingham
  NCT07102043updated 2026-06-01
• Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
  Active not recruiting · Phase 3 · Interventional · 297 enrolled · Vertex Pharmaceuticals Incorporated
  NCT05331183updated 2026-05-29
• A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
  Active not recruiting · Phase 1 · Phase 2 · Interventional · 26 enrolled · Vertex Pharmaceuticals Incorporated
  NCT05668741updated 2026-05-22
• Trikafta Exercise Study in Cystic Fibrosis
  Recruiting · Observational · 20 enrolled · University of British Columbia
  NCT05279040updated 2026-05-18
• Sinus Disease in Young Children With Cystic Fibrosis
  Recruiting · Observational · 80 enrolled · University of California, Los Angeles
  NCT06191640updated 2026-05-11
• The PROMISE Pediatric Study 6 to 11 Years Old
  Completed · Observational · 125 enrolled · Nicole Hamblett
  NCT04613128updated 2026-04-27
• Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis
  Recruiting · Early phase 1 · Interventional · 20 enrolled · University of Alabama at Birmingham
  NCT03439865updated 2026-04-22

CPIC-curated drug–gene pairs for Ivacaftor. Levels describe the strength of curated evidence and guideline status — never a recommendation to test or to adjust therapy.

• CFTRCPIC AClinPGx 1AFDA label: Testing Required
  CFTR and Ivacaftor

Frequently asked questions

How does Ivacaftor work?

Ivacaftor is a potentiator of the CFTR protein. The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs.

What is Ivacaftor used for?

According to FDA labeling, Ivacaftor carries indications including: KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . 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.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Ivacaftor?

Ivacaftor is classified as Other respiratory system products, Cystic Fibrosis Transmembrane Conductance Regulator Potentiator, Chloride Channel Activation Potentiators, Cytochrome P450 2C9 Inhibitors, Cytochrome P450 3A Inhibitors, P-Glycoprotein Inhibitors.

What are the brand names for Ivacaftor?

Ivacaftor is marketed under brand names including Kalydeco, ORKAMBI.

What are the contraindications for Ivacaftor?

Ivacaftor labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.