Dosage and Administration

Dosage and Administration

KALYDECO® (ivacaftor) is supplied as tablets (150 mg) for people 6 years and older and as oral granules (50 mg and 75 mg packets) for people 2 to less than 6 years of age. Please click here for Dosage and Administration information for KALYDECO oral granules.

Oral dosing in a 150 mg tablet1

KALYDECO 150 mg tablets, the first cystic fibrosis treatment for patients who have a G551D mutation

All images are not to scale.

  • The recommended dose of KALYDECO for both adults and pediatric patients age 6 years and older is one 150 mg tablet taken orally every 12 hours (300 mg total daily dose) with fat-containing food
    • Food containing grapefruit or Seville oranges may increase exposure of ivacaftor and should be avoided during treatment with KALYDECO
    • Fat-containing food enhanced absorption 2- to 4-fold; therefore, KALYDECO should be taken with fat-containing food

Reduce dose in patients with moderate and severe hepatic impairment and when co-administered with drugs that are moderate or strong CYP3A inhibitors. Please see the Dose Adjustments page for more information

Patients should continue taking their prescribed CF therapies with KALYDECO1

Administration with fat-containing food11

Here are some examples of fat-containing meals and snacks:

Examples of some fat-containing food

This list is not intended to be exhaustive.

Important Safety Information—
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

Please click here for additional Important Safety Information.

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 2 years and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.

KALYDECO is indicated for the treatment of CF in patients age 2 years and older who have an R117H mutation in the CFTR gene.

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.

Limitation of Use:

KALYDECO is not effective in patients with CF who are homozygous for the F508del mutation in the CFTR gene.

Important Safety Information

Transaminase (ALT or AST) Elevations

  • Elevated transaminases have been reported in patients with CF receiving KALYDECO. Transaminase elevations were more common in patients with a history of transaminase elevations or in patients who had abnormal transaminases at baseline. It is recommended that ALT and AST be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. For patients with a history of transaminase elevations, more frequent monitoring of liver function tests should be considered
  • Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN). Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO dosing

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 2 years of age have not been studied. The use of KALYDECO in children under the age of 2 years 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

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 (24% vs 16%), oropharyngeal pain (22% vs 18%), upper respiratory tract infection (22% vs 14%), nasal congestion (20% vs 15%), abdominal pain (16% vs 13%), nasopharyngitis (15% vs 12%), diarrhea (13% vs 10%), rash (13% vs 7%), nausea (12% vs 11%), and dizziness (9% vs 1%)
  • The safety profiles for patients with a G1244E, G1349D, G178R, G551S, G970R, S1251N, S1255P, S549N, or S549R mutation enrolled in Trial 4, for patients with an R117H mutation enrolled in Trial 5, and for patients ages 2 to less than 6 years enrolled in
    Trial 6 were similar to that observed in Trials 1 and 2

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

References: 1. KALYDECO (ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; March 2015. 2. Ramsey BW, Davies J, McElvaney NG, et al; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672. 3. Davies JC, Wainwright CE, Canny GJ, et al; VX08-770-103 (ENVISION) Study Group. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med. 2013;187(11):1219-1225. 4. Zielenski J. Genotype and phenotype in cystic fibrosis. Respiration. 2000;67(2):117-133. 5. Welsh MJ, Ramsey BW, Accurso F, Cutting GR. Cystic fibrosis: membrane transport disorders. In: Valle D, Beaudet A, Vogelstein B, et al, eds. The Online Metabolic & Molecular Bases of Inherited Disease. The McGraw-Hill Companies, Inc; 2004:part 21, chap 201. http://www.ommbid.com. Accessed February 25, 2016. 6. Welsh MJ, Smith AE. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell. 1993;73(7):1251-1254. 7. Orenstein DM, Spahr JE, Weiner DJ. Cystic Fribrosis: A Guide for Patient and Family. 4th ed. Philadelphia, PA: Lippincott Williams & Wilkins; 2012. 8. Yu H, Burton B, Huang C-J, et al. Ivacaftor potentiation of multiple CFTR channels with gating mutations. J Cyst Fibros. 2012;11(3):237-245.
9. US CF Foundation, Johns Hopkins University. The Hospital for Sick Children. The Clinical and Functional TRanslation of CFTR (CFTR2). http://www.cftr2.org/index.php. Accessed February 25, 2016. 10. Berwouts S, Morris MA, Girodon E, Schwarz M, Stuhrmann M, Dequeker E. Mutation nomenclature in practice: findings and recommendations from the cystic fibrosis external quality assessment scheme. Hum Mutat. 2011;32(11):1197-1203. 11. US Department of Agriculture, Agricultural Research Service, Nutrient Data Laboratory Web site. ndb.nal.usda.gov/. Accessed February 25, 2016.

The information contained in this section of the site is intended for US Healthcare Professionals only.
Are you a US Healthcare Professional?

Yes
No

You are now leaving www.kalydeco.com. Vertex Pharmaceuticals Incorporated is
not responsible for the content of the site you are about to visit.
Please click "OK" to continue or "Cancel" to go back to www.kalydeco.com

OK
Cancel