Inborn errors of metabolism:

Indications for: KALYDECO

Treatment of cystic fibrosis (CF) in patients ≥4mos who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.

Adults and Children:

<4mos, 4–<6mos (with hepatic impairment and/or on concomitant moderate or strong CYP3A inhibitors): not recommended. Oral granules should be mixed with 1 tsp (5mL) of soft-food or liquid (eg, yogurt, applesauce, milk, or juice) and completely consumed. Take with fat-containing food (eg, eggs, butter, peanut butter, cheese pizza, dairy products). 4–<6mos (≥5kg): 25mg packet every 12hrs. 6mos–<6yrs (5–<7kg): 25mg packet every 12hrs; (7–<14kg): 50mg packet every 12hrs; (≥14kg): 75mg packet every 12hrs. ≥6yrs: 150mg tab every 12hrs. Concomitant CYP3A inhibitors (≥6mos): reduce to twice weekly (strong inhibitors) or once daily (moderate inhibitors). Hepatic impairment (≥6mos): reduce to once daily for moderate impairment, once daily or less frequently if severe.

KALYDECO Warnings/Precautions:

If genotype is unknown, use an FDA-cleared CF mutation test to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions. Assess ALT/AST levels prior to initiating therapy, every 3 months during the first year of treatment, and annually thereafter. History of ALT/AST elevations: monitor LFTs more frequently. If increased ALT/AST levels develop, monitor closely until abnormalities resolved. Interrupt dosing if ALT/AST is >5xULN; after resolution, consider restarting. Perform baseline and follow-up eye exams. Hepatic impairment. Severe renal impairment or ESRD. Pregnancy. Nursing mothers.

KALYDECO Classification:

Cystic fibrosis transmembrane conductance regulator (CFTR) potentiator.

KALYDECO Interactions:

Potentiated by strong CYP3A inhibitors (eg, ketoconazole, itraconazole, posaconazole, voriconazole, telithromycin, clarithromycin) and moderate CYP3A inhibitors (eg, fluconazole, erythromycin); see Adults and Children. May be potentiated by grapefruit and Seville oranges; avoid. Antagonized by strong CYP3A inducers (eg, rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, St. John’s Wort); use not recommended. Potentiates CYP3A and/or P-gp substrates (eg, digoxin, cyclosporine, tacrolimus); monitor.

Adverse Reactions:

Headache, oropharyngeal pain, upper respiratory tract infection, nasal congestion, abdominal pain, nasopharyngitis, diarrhea, rash, nausea, dizziness; non-congenital lens opacities/cataracts.

Generic Drug Availability:


How Supplied:

Tabs—56, 60; Oral granules—56